2019s Allen Distinguished Investigators will focus on the mysteries of our cells – GeekWire

Biomedical researcher Samantha Morris, shown here in her lab at Washington University School of Medicine in St. Louis, is one of the newly named Allen Distinguished Investigators. This award is enabling us to take a big risk in our arena by generating a completely new technology, one which will be useful to the scientific community. Thats really exciting for us, she said. (Washington University in St. Louis Photo)

The Paul G. Allen Frontiers Group, a division of Seattles Allen Institute, is making a total of $7.5 million in awards to its latest class of five biomedical researchers.

The themes for this years Allen Distinguished Investigators focus on stem cell therapies and single-cell interactions in their native environments.

The field of stem cell biology has the potential to change how we treat diseases by helping precision medicine, and theres so much we still dont understand about the interplay between cells in living tissues or organs, Kathy Richmond, director of the Frontiers Group, said today in a news release.

Our 2019 Allen Distinguished Investigators are pushing their fields in these two areas, through new technology development, probing pivotal interactions in the body that cause health to fail, and generating creative new stem cell models that will improve our understanding of different human diseases, she said.

The late Microsoft co-founder Paul Allen gave the Allen Distinguished Investigator program its start in 2010 as a way to support significant early-stage research thats less likely to receive grants from traditional sources. This years selections bring the roster to a total of 74 researchers, including 13 from the University of Washington.

Each of the investigators will receive $1.5 million in support for their projects over three years. Heres a rundown on the Class of 2019:

Samantha Morris of Washington University in St. Louis aims to create a blueprint of cell identity that will enable researchers to improve the way they generate different kinds of cells from human stem cells.

Joshua Rabinowitz of Princeton University will lead a team developing new technologies to study metabolites, the molecules that result from our bodies conversion of food into energy, as well as metabolic activity in single cells from mouse and human tissue.

Clive Svendsen of Cedars-Sinai Medical Center will use stem cells to model how interactions between the gut microbiome and the brain might influence neuron death in patients with Parkinsons disease.

Savas Tay and his colleagues at the University of Chicago are looking into the roots of Crohns disease by combining the study of gene expression with single-cell measurements of proteins and protein complexes, using samples of healthy and diseased gut tissue.

James Wells and his colleagues of Cincinnati Childrens Hospital Medical Center will use stem cells to study maladies that affect enteroendocrine cells, which sense nutrients from the food we eat and then control how those nutrients are processed in the intestines.

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2019s Allen Distinguished Investigators will focus on the mysteries of our cells - GeekWire

Cellular Biomedicine Group Reports Third Quarter of 2019 Financial Results and Business Highlights – P&T Community

NEW YORK and SHANGHAI, Nov. 6, 2019 /PRNewswire/ --Cellular Biomedicine Group Inc.(NASDAQ: CBMG) ("CBMG" or the "Company"), a biopharmaceutical firm engaged in the drug development of immunotherapies for cancer and stem cell therapies for degenerative diseases, today reported its financial results and business highlights for the third quarter of 2019.

"During the third quarter of 2019, we made great strides in both corporate and clinical progress. We started our U.S. expansion for research and clinical development in a new 22,000 square foot facility in Rockville, Maryland in October 2019. This milestone will allow us to foster strategic partnerships, develop new innovations and support continued development of CBMG's cell therapy-based immune-oncology assets that have shown promise in early proof-of-concept trials in China," said Tony (Bizuo) Liu, Chief Executive Officer for the Company.

"We also had continued progress on the clinical side, with the initiation of our Phase II clinical trial in China of AlloJoin therapy for knee osteoarthritis (KOA). Additionally, our autologous stem cell therapy program for KOA, ReJoin, was accepted by the NMPA in China to begin a Phase II clinical trial. We are excited about our regenerative medicine programs as we are currently the only company that has received two clinical trial acceptances for any stem cell program in China."

Mr. Liu continued, "Presentations of our pre-clinical and clinical data at upcoming medical conferences later this year will demonstrate continued focus on our immune-oncology pipeline and we are proud to provide an update of our commitment to cancer immunotherapy."

Third Quarter 2019 and Other Recent Corporate Developments

Upcoming Clinical and Preclinical Presentations:

Financial Results for the Third Quarter of 2019

Conference Call and Webcast InformationThe Company will host a conference call and webcast with the investment community on Wednesday, November 6th at 4:30 p.m. Eastern Time featuring remarks by Tony Liu, Executive Director, CEO and CFO of CBMG.

Live Call:

Toll-Free: 1-855-327-6838

International: 1-604-235-2082

Webcast:

http://public.viavid.com/index.php?id=136796

Replay:

Toll-Free: 1-844-512-2921

International: 1-412-317-6671

Conference ID: 10007976

(Available approximately two hours after the completion of the live call until 11:59 p.m. ET on November 20, 2019)

About Cellular Biomedicine GroupCellular Biomedicine Group, Inc. (NASDAQ: CBMG) develops proprietary cell therapies for the treatment of cancer and degenerative diseases. It conducts immuno-oncology and stem cell clinical trials in China using products from its integrated GMP laboratory. The Company's GMP facilities in China, consisting of twelve independent cell production lines, are designed and managed according to both China and U.S. GMP standards. Its Shanghai facility includes a "Joint Laboratory of Cell Therapy" with GE Healthcare and a "Joint Cell Therapy Technology Innovation and Application Center" with Thermo Fisher Scientific, which partnerships focus on improving manufacturing processes for cell therapies. CBMG currently has ongoing CAR-T Phase I clinical trials in China. The China NMPA (formerly CFDA) accepted the Company's IND application for a Phase II trial for AlloJoin, CBMG's "Off-the-Shelf" allogenic haMPC therapy for the treatment of Knee Osteoarthritis (KOA), and the Company's IND application for a Phase II trial for ReJoin autologous haMPC therapy for the treatment of KOA. CBMG is included in the broad-market Russell 3000 Index and the small-cap Russell 2000 Index, and the Loncar China BioPharma index. To learn more about CBMG, please visit http://www.cellbiomedgroup.com.

Forward-Looking StatementsStatements in this press release relating to plans, strategies, trends, specific activities or investments, and other statements that are not descriptions of historical facts and may be forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. Forward-looking information is inherently subject to risks and uncertainties, and actual results could differ materially from those currently anticipated due to a number of factors, which include those regarding our ability to implement our plans, strategies and objectives for future operations, including regulatory approval of our IND applications, our plan to configure part of our Shanghai facility with GE Healthcare's FlexFactory platform, our ability to execute on our obligations under the terms of our licensing and collaboration arrangement with Novartis, our ability to execute on proposed new products, services or development thereof, results of our clinical research and development, regulatory infrastructure governing cell therapy and cellular biopharmaceuticals, our ability to enter into agreements with any necessary manufacturing, marketing and/or distribution partners for purposes of commercialization, our ability to seek intellectual property rights for our product candidates, competition in the industry in which we operate, overall market conditions, any statements or assumptions underlying any of the foregoing and other risks detailed from time to time in CBMG's reports filed with the Securities and Exchange Commission, Annual Reports on Form 10-K, Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K. Forward-looking statements may be identified by terms such as "may," "will," "expects," "plans," "intends," "estimates," "potential," or "continue," or similar terms or the negative of these terms. Although CBMG believes the expectations reflected in the forward-looking statements are reasonable, they cannot guarantee that future results, levels of activity, performance or achievements will be obtained. CBMG does not have any obligation to update these forward-looking statements other than as required by law.

For more information, please contact:

Company Contact:Derrick C. LiHead of Strategy and Investor Relations, CBMGPhone: 917-717-0994Email: derrick.li@cellbiomedgroup.com

Investor Contact:Valter Pinto / Allison SossKCSA Strategic CommunicationsPhone: 212-896-1254 / 212-896-1267Email: cellbiomed@kcsa.com

CELLULAR BIOMEDICINE GROUP, INC.

CONDENSED CONSOLIDATED BALANCE SHEETS

(UNAUDITED)

September 30,

December 31,

2019

2018

Assets

Cash and cash equivalents

$29,035,677

$52,812,880

Restricted cash

17,000,000

-

Accounts receivable, less allowance for doubtful accounts of nil and $94,868 as of September 30, 2019 and December 31, 2018, respectively

-

787

Other receivables

591,271

101,909

Prepaid expenses

1,589,479

1,692,135

Total current assets

48,216,427

54,607,711

Investments

240,000

240,000

Property, plant and equipment, net

19,856,287

15,193,761

Right of use

14,298,613

15,938,203

Goodwill

7,678,789

7,678,789

Intangibles, net

7,521,523

7,970,692

Long-term prepaid expenses and other assets

7,640,535

5,952,193

Total assets

$105,452,174

$107,581,349

Liabilities and Stockholders' Equity

Liabilities:

Short-term debt

$14,138,419

$-

Accounts payable

5,686,023

422,752

Accrued expenses

1,477,174

1,878,926

Taxes payable

28,625

28,950

Other current liabilities

4,526,594

5,710,578

Total current liabilities

25,856,835

8,041,206

Other non-current liabilities

12,545,245

14,321,751

Total liabilities

38,402,080

22,362,957

Stockholders' equity:

Preferred stock, par value $.001, 50,000,000 shares authorized; none issued and outstanding as of September 30, 2019 and December 31, 2018, respectively

-

-

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Cellular Biomedicine Group Reports Third Quarter of 2019 Financial Results and Business Highlights - P&T Community

Cost is a major challenge in stem cells therapy: Dr Na’eem Sadiq – ETHealthworld.com

Shahid Akhter, editor, ETHealthworld spoke to Dr Na'eem Sadiq, Medical Director, PLEXUS NEURO and STEM CELL RESEARCH CENTER, Bengaluru to know more about stem cell therapy and the challenges associated with it.

STEM CELL : TRENDSStem cell is a word which evokes lot of responses both positive and negative. Few people know that the origin was in the 1800s. The very first bone marrow transplant happened in the year 1968 and then subsequently stem cells have been used for various diseases and much more in blood cancer. They have also been used in chronic neurological disorders, autoimmune disorders and sports injuries.Globally, its all over the world such as in the US, Canada, Germany, China, Ukraine and of course in India as well. In India there are lots of centers and states who have been practicing stem cell technology for quite some time.

STEM CELLS : MARKETThe market is growing since stems cells promise hope for those who have lost hope, where there is no viable treatment and proper cure available for lots of diseases. Stem cells is emerging as a champion for all these people. It was much more available internationally and in the last decade India has taken up.

PLEXUS NEURO AND STEM CELL RESEARCH CENTER- JOURNEYI have been practicing in the field of neuroscience for the last 30 years. Neurosciences is a field where you see patients suffering from chronic diseases. I have been in this field right from early 90s and have been seeing trends changing, but when it comes to neurodegenerative disorders such as Parkinsons, ALS, Multiple Sclerosis, billions of dollars have been spent, new treatment modalities have been found, but nothing has been found to be successful.

We have very strict and rigid eligibility criteria. Once the patient approaches us, we subject the patient to a thorough clinical examination, which lasts anywhere between 2- 3 hours. Once we find that the patient is clinically treatable, or that the patient can be helped, then we subject the patient to other investigations.

The other major difference we have at Plexus is that we do not do only stem cells. Stem cell therapy is a part of our complete regenerative rehabilitation. The program starts after we do the transplant. The patient undergoes rigorous rehabilitation, which includes the entire gamete of practices such as physical therapy, occupational therapy, hand splinting, cognitive rehabilitation therapy, cognitive behavior therapy, speech therapy etc.

We customize and provide a tailor made program as per the patient's needs, with a goal once the patient joins the program and almost all the patients who are in the program get more than what we had aimed at achieving. At the end of the program we evaluate the goals and find that every single patient achieves them. We train the patients as to what they need to do once they finish the program and insist on regular follow up.

We have a team of learned scientists who are all qualified from the UK and our research is ongoing. We are working exclusively in the field of neurosciences to get the best quality of cells and to make it very affordable. Research is on and our data is huge, we will be publishing the results very soon.

PLEXUS : FUTURE PLANSWe have a complete state of the art rehabilitation center where we have some of the best therapists in the world working with us. In fact a few months back we launched one of its kind, Sensory Gym at Plexus and now we have started virtual and augmented reality.

In the last 4-5 years we have received more than 75 national and international awards and we stand as one of the leading regenerative rehabilitation centers not only in India, but in Asia.Our endeavor here is to make the treatment the best possible, to make the cells much more advanced, affordable to also provide the treatment in the shortest possible time.

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Cost is a major challenge in stem cells therapy: Dr Na'eem Sadiq - ETHealthworld.com

Cardon Children’s is proud of its 10-year history – East Valley Tribune

Ten years ago this month, Banner Health and our East Valley community celebrated, with great fanfare, the opening of Cardon Childrens Medical Center, a beautiful seven-story hospital in Mesa built through the eyes of a child.

Since the opening, Cardon Childrens has had the honor of caring for thousands of kids. In fact, we have had more than 105,000 pediatric patient admissions and nearly 405,000 patient visits to our pediatric emergency room over the past 10 years.

In 2017, the hospital was designated a Level 1 Trauma Center for pediatric patients, capable of handling the most severe cases.

Many people wont remember, but, prior to Cardon Childrens opening, Banner Childrens Hospital was a single wing inside Banner Desert Medical Center.

Excellent pediatric care was provided there, but the space was not optimal, nor was it adequate.

It was not unusual for pediatric patients to be sent out of the area sometimes out of state for a needed pediatric hospital bed.

There was a pediatric hospital capacity shortage here in the early 2000s, particularly during peak flu and RSV seasons.

Banner Health saw the need and invested more than $300 million to ensure that children in the East Valley, around the state and, even in neighboring states, had access to the best pediatric care possible in a state-of-the-art facility.

Over the years and recently, Banner Childrens has recruited some of the very best pediatric specialists to help us carry out our important mission. Recent additions include:

Dr. Gordon Cohen, medical director of the Congenital Heart Surgery Center, is an internationally recognized leader in pediatric cardiothoracic surgery. He led Seattle Childrens Hospital from a nonsurgical cardiology program to a top program for Cardiology and Cardiac Surgery.

Dr. Emmanuel Katsanis, chief of Banner Childrens/U of A state-wide Pediatric Blood and Cancer Program. He also is internationally recognized for his research in transplant immunology, and is the leader of the Banner/University of Arizona Stem Cell Transplant Program for adults and children.

Dr. David Moss, a pediatric neurosurgeon who is considered one of the most skilled, talented and knowledgeable neurosurgeons in the Southwestern U.S.

Dr. Sandra Buttram, a pediatric intensivist with specialized training and expertise in neuro-critical care and ECMO.

Dr. Hiep Nguyen, an internationally recognized leader in pediatric urology and, in particular, robotic surgery.

Dr. Mark Joseph, medical director of Pediatric Nephrology, a nationally recognized leader in his field.

Ten years seems to go by in the blink of an eye.

It seems like only yesterday that we were welcoming our first patients, and weve celebrated many important milestones since then.

We are proud the hospital has grown in stature and prominence, but, importantly, we are also proud to have remained a comforting and caring place for sick and injured children in their time of need.

Our greatest pride is in our staff the dedicated physicians, nurses and other medical caregivers who give selflessly day in and day out. Were not just celebrating a building when we mark this 10-year anniversary Were celebrating our Cardon Childrens pediatric professionals, past and present, because without them we would not be where we are today.

On behalf of everyone at Cardon Childrens, I want to thank the community for putting your trust in us when your precious children need medical care. We do not take that responsibility lightly, and we understand you hold us to the highest level of professionalism. We consider it an honor to serve, and look forward to the next 10 years and beyond.

Laura Robertson is chief executive officer, Cardon Childrens Medical Center and Banner Desert Medical Center.

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Cardon Children's is proud of its 10-year history - East Valley Tribune

Mustang Bio Announces MB-107 Lentiviral Gene Therapy and MB-106 CD20-Targeted CAR T Data Selected for Presentations at 61st American Society of…

St. Jude Childrens Research Hospital and the National Institutes of Health to present updated MB-107 clinical data for the treatment of X-linked severe combined immunodeficiency

Fred Hutchinson Cancer Research Center to present overview of ongoing MB-106 Phase 1/2 clinical trial

NEW YORK, Nov. 06, 2019 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. (Mustang) (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, announced today that updated Phase 1/2 clinical data for MB-107 lentiviral gene therapy for X-linked severe combined immunodeficiency (XSCID) have been selected for oral and poster presentations at the 61st American Society of Hematology (ASH) Annual Meeting. ASH will be held December 7-10, 2019, at the Orange County Convention Center in Orlando, FL.

MB-107 is currently being assessed in two Phase 1/2 clinical trials for XSCID: the first in newly diagnosed infants under the age of two at St. Jude Childrens Research Hospital, UCSF Benioff Childrens Hospital and Seattle Childrens Hospital and the second in patients over the age of two who have received prior hematopoietic stem cell transplantation at the National Institutes of Health. Positive Phase 1/2 clinical data from the trial for infants under the age of two were published in the New England Journal of Medicine in April 2019 and positive Phase 1/2 clinical data from the trial in patients over the age of two were published in Science Translational Medicine in April 2016. The U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to MB-107 for the treatment of XSCID in August 2019.

Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, We are extremely pleased that additional clinical data on MB-107, a lentiviral gene therapy for the treatment of XSCID, will be presented in oral and poster sessions at the 2019 ASH Annual Meeting. The curative potential of MB-107 based on previously announced compelling Phase 1/2 data is impressive, and we look forward to working with St. Jude and NIH to advance the development of this important treatment option.

Details of the MB-107 presentations are as follows.

Oral Presentation:Title: Enhanced Transduction Lentivector Gene Therapy for Treatment of Older Patients with X-Linked Severe Combined ImmunodeficiencySession: 801. Gene Therapy and Transfer: Gene Therapies for Non-Malignant DisordersAbstract Number: 608Date and Time: Monday, December 9, 2019, 7:15 a.m. ET Location: Orange County Convention Center, Valencia BC (W415BC)Presenter: Harry Malech, M.D., Laboratory of Clinical Immunology and Microbiology, NIAID, NIH, Bethesda, MD, USA

Poster Presentation:Title: Lentiviral Gene Therapy with Low Dose Busulfan for Infants with X-SCID Results in the Development of a Functional Normal Immune System: Interim Results of an Ongoing Phase I/II Clinical StudySession: 801. Gene Therapy and Transfer: Poster IAbstract Number: 2058Date and Time: Saturday, December 7, 2019, 5:30-7:30 p.m. ETLocation: Orange County Convention Center, Hall BPresenter: Ewelina Mamcarz, M.D., Department of Bone Marrow Transplantation and Cellular Therapy, St. Jude Childrens Research Hospital, Memphis, TN, USA

In addition, Mustangs collaborator Fred Hutchinson Cancer Research Center will present a poster about the ongoing Phase 1/2 clinical trial investigating the safety and efficacy of MB-106 CD20-targeted CAR T for high-risk B-cell non-Hodgkin lymphomas.

Details of the MB-106 presentation are as follows.

Poster Presentation:Title: CD20 Targeted CAR-T for High-Risk B-Cell Non-Hodgkin LymphomasSession: 704. Immunotherapies: Poster IIAbstract Number: 3235 Date and Time: Sunday, December 8, 2019, 6-8 p.m. ETLocation: Orange County Convention Center, Hall BPresenter: Mazyar Shadman, M.D., M.P.H., Fred Hutchinson Cancer Research Center, Seattle, WA, USA

Copies of the abstracts can be viewed online through the ASH website at http://www.hematology.org.

About Mustang BioMustang Bio, Inc. (Mustang) is a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases. Mustang aims to acquire rights to these technologies by licensing or otherwise acquiring an ownership interest, to fund research and development, and to outlicense or bring the technologies to market. Mustang has partnered with top medical institutions to advance the development of CAR T and CRISPR/Cas9-enhanced CAR T therapies across multiple cancers, as well as a lentiviral gene therapy for XSCID. Mustang is registered under the Securities Exchange Act of 1934, as amended, and files periodic reports with the U.S. Securities and Exchange Commission. Mustang was founded by Fortress Biotech, Inc. (NASDAQ: FBIO). For more information, visit http://www.mustangbio.com.

ForwardLooking Statements This press release may contain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, each as amended. Such statements include, but are not limited to, any statements relating to our growth strategy and product development programs and any other statements that are not historical facts. Forward-looking statements are based on managements current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock value. Factors that could cause actual results to differ materially from those currently anticipated include: risks relating to our growth strategy; our ability to obtain, perform under and maintain financing and strategic agreements and relationships; risks relating to the results of research and development activities; risks relating to the timing of starting and completing clinical trials; uncertainties relating to preclinical and clinical testing; our dependence on third-party suppliers; our ability to attract, integrate and retain key personnel; the early stage of products under development; our need for substantial additional funds; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law.

Company Contacts:Jaclyn Jaffe and William BegienMustang Bio, Inc.(781) 652-4500ir@mustangbio.com

Investor Relations Contact:Daniel FerryLifeSci Advisors, LLC(617) 430-7576daniel@lifesciadvisors.com

Media Relations Contact:Tony Plohoros6 Degrees(908) 940-0135tplohoros@6degreespr.com

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Mustang Bio Announces MB-107 Lentiviral Gene Therapy and MB-106 CD20-Targeted CAR T Data Selected for Presentations at 61st American Society of...

Celebrating Cancer Heroes will recognize survivors at event in Somers – Reminder Publications

SOMERS Six area cancer survivors will be honored at Celebrating Cancer Heroes, an event that benefits Survivor Journeys, an organization that provides support for cancer survivors and awareness of cancer survivorship.

The individuals being recognized are Luke Bradley of South Hadley, Liz Wilson Greer of Springfield, Candy Oyler of Southwick, Heidi Huhn-Partain of Suffield, CT., Megan Rothschild of Northampton and Chris Thibault of Springfield.

These heroes have inspired their community and others as they persevered through the challenges of their diagnosis with a brave and determined spirit. Many of our heroes have volunteered in the community or inspired others to volunteer, the organization stated.

More than 60 people nominated their heroes. The criteria for a hero is someone who has gone through their cancer struggles acts as a beacon to people who are still struggling, said Jay Burton, the founder and president of Survivor Journeys.

The Nov. 21 event begins at 6 p.m. and will be a social fundraising evening at Monticello, located at 732 Hall Hill Rd., in Somers, CT. The mansion was built by S. Prestley Blake, co-founder of the Friendlys restaurant company. It is a replica of the eighteenth century home of Thomas Jefferson by the same name. Chez Josef will be catering the business casual evening.

Rich Tettemer, an anchor from WWLP-TV 22 News, will be the emcee for the evening and retired Western Mass News anchor Dave Madsen will be a guest speaker.

Tickets are $85 per person and $25 for individuals 18 and under. A limited number of tables of 10 are available for $750. Tickets can be purchased at survivorjourneys.org or by calling 276-6100. Survivor Journeys is hoping to raise $25,000 from the night. Sponsors of the celebration include Johnson Memorial Medical Center in Stafford Springs, Saint Francis Medical Center in Hartford, and Trinity Health of New England, as well as WWLP-TV 22/CW.

Survivor Journeys is a non-profit organization founded in March 2015. Burton said people are cancer survivors the moment theyre diagnosed, because you go into survival mode. He defines cancer survivorship as living before, during and after cancer treatment.

Survivor Journeys offers free access to more than 10,000 mentors to assist and support cancer patients with emotional, psychological and practical needs after their diagnosis. These trained cancer survivors have helped people from as far away as Arizona, Burton said.

There are free support groups for breast cancer, blood cancer, head and neck cancer, non-specified cancer and a caregiver group. The groups are located as far south as Enfield, CT., and as far north as Holyoke.

The organization also provides an animal therapy program and Cancer Survivorship 101, an annual workshop, designed to educate survivors, caregivers, loved ones about the factors that play a role before, during and after cancer treatment.

Burton, who is a primary care doctor in Enfield, CT., feels so strongly about cancer survivor care because he, himself, is a survivor of acute myeloid leukemia. In 2010, he had a stem cell transplant to treat the disease. Because of this, he is in a position to see both sides of the problem.

Our hospitals havent seen this as a priority, Burton said. He said patients daily lives and support dont get addressed when they go to see their oncologist. He noted that this problem is not unique to New England, but is nationwide.

Burton related to Reminder Publishing a conversation he had had with a doctor in New York City. He had asked the doctor about how people got to his office. He said that patients who are sick have to stand out in the elements for a bus or climb stairs to use the subway. The doctor had never considered it, Burton said.

Burton said there needs to be a stronger focus on psychosocial support for patients and their caregivers.

I know the limitations of our medical system, said Burton, but it doesn't mean we don't strive to make it better.

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Celebrating Cancer Heroes will recognize survivors at event in Somers - Reminder Publications

Fate Therapeutics Reports Third Quarter 2019 Financial Results and Highlights Operational Progress – Yahoo Finance

First Patients Treated with FT516, an Off-the-Shelf NK Cell Cancer Immunotherapy for AML and for B-cell Lymphoma in Combination with Rituximab

Received FDA Clearance of IND Application for FT596, an Off-the-Shelf, Multi-Antigen Targeted CAR NK Cell Product Candidate

Opened State-of-the-art cGMP Facility Dedicated to Manufacturing iPSC-derived Cell Therapies

$303 Million in Cash & Short-term Investments as of September 30, 2019 following Completion of $173 Million Common Stock Offering

SAN DIEGO, Nov. 05, 2019 (GLOBE NEWSWIRE) -- Fate Therapeutics, Inc. (FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, today reported business highlights and financial results for the third quarter ended September 30, 2019.

We achieved several significant clinical milestones over the past three months including treating the first patients with FT516, the first-ever engineered iPSC-derived cellular immunotherapy, and securing FDA clearance to initiate clinical investigation of FT596, the first-ever cellular immunotherapy engineered to express three active anti-tumor modalities. We also successfully opened our new cGMP facility specifically designed to enable consistent, large-scale, and cost-effective manufacture of allogeneic NK cell and CAR-T cell products using clonal master iPSC lines as a starting cell source, said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. We look forward to the ASH annual meeting in December, where we have had six abstracts accepted and will be sharing our first-in-human insights into the clinical safety and tolerability of FT500, the first-ever iPSC-derived cell therapy to be administered off-the-shelf in multiple doses over multiple cycles. With the completion of our recent common stock offering in September, we are well-positioned to generate clinical data across our iPSC-derived, cell-based cancer immunotherapy pipeline in 2020.

Clinical Programs

Corporate Highlights

Third Quarter 2019 Financial Results

Today's Conference Call and WebcastThe Company will conduct a conference call today, Tuesday, November 5, 2019 at 5:00 p.m. ET to review financial and operating results for the quarter ended September 30, 2019. In order to participate in the conference call, please dial 877-303-6235 (domestic) or 631-291-4837 (international) and refer to conference ID 4748666. The live webcast can be accessed under "Events & Presentations" in the Investors & Media section of the Company's website at http://www.fatetherapeutics.com. The archived webcast will be available on the Company's website beginning approximately two hours after the event.

About Fate Therapeutics iPSC Product PlatformThe Companys proprietary induced pluripotent stem cell (iPSC) product platform enables mass production of off-the-shelf, engineered, homogeneous cell products that can be administered with multiple doses to deliver more effective pharmacologic activity, including in combination with cycles of other cancer treatments. Human iPSCs possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body. The Companys first-of-kind approach involves engineering human iPSCs in a one-time genetic modification event and selecting a single engineered iPSC for maintenance as a clonal master iPSC line. Analogous to master cell lines used to manufacture biopharmaceutical drug products such as monoclonal antibodies, clonal master iPSC lines are a renewable source for manufacturing cell therapy products which are well-defined and uniform in composition, can be mass produced at significant scale in a cost-effective manner, and can be delivered off-the-shelf for patient treatment. As a result, the Companys platform is uniquely capable of overcoming numerous limitations associated with the production of cell therapies using patient- or donor-sourced cells, which is logistically complex and expensive and is subject to batch-to-batch and cell-to-cell variability that can affect clinical safety and efficacy. Fate Therapeutics iPSC product platform is supported by an intellectual property portfolio of over 250 issued patents and 150 pending patent applications.

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About FT500FT500 is an investigational, universal, off-the-shelf natural killer (NK) cell cancer immunotherapy derived from a clonal master induced pluripotent stem cell (iPSC) line. The product candidate is being investigated in an open-label, multi-dose Phase 1 clinical trial for the treatment of advanced solid tumors (clinicaltrials.gov ID number NCT03841110). The study is designed to assess the safety and activity of three once-weekly doses of FT500 as a monotherapy and in combination with one of three FDA-approved immune checkpoint inhibitor (ICI) therapies nivolumab, pembrolizumab or atezolizumab in patients that have failed prior ICI therapy. Despite the clinical benefit conferred by approved ICI therapy against a variety of tumor types, these therapies are not curative and, in most cases, patients either fail to respond or progress on these agents. One common mechanism of resistance to ICI therapy is associated with loss-of-function mutations in genes critical for antigen presentation. A potential strategy to overcome resistance is through the administration of allogeneic NK cells, which have the inherent capability to recognize and directly kill tumor cells with these mutations.

About FT516FT516 is an investigational, universal, off-the-shelf natural killer (NK) cell cancer immunotherapy derived from a clonal master induced pluripotent stem cell (iPSC) line engineered to express a novel high-affinity 158V, non-cleavable CD16 Fc receptor, which has been modified to prevent its down-regulation and enhance its binding to tumor-targeting antibodies. The product candidate is being investigated in an open-label, multi-dose Phase 1 clinical trial as a monotherapy for the treatment of acute myeloid leukemia and in combination with CD20-directed monoclonal antibodies for the treatment of advanced B-cell lymphoma (clinicaltrials.gov ID number NCT04023071). CD16 mediates antibody-dependent cellular cytotoxicity (ADCC), a potent anti-tumor mechanism by which NK cells recognize, bind and kill antibody-coated cancer cells. CD16 occurs in two variants, either with high (158V) or low (158F) affinity for the Fc domain of IgG1 antibodies. Numerous clinical studies with FDA-approved tumor-targeting antibodies, including rituximab, trastuzumab and cetuximab, have demonstrated that patients homozygous for the 158V variant, which is present in only about 15% of patients, have improved clinical outcomes. In addition, ADCC is dependent on NK cells maintaining active levels of CD16 expression, and the expression of CD16 on NK cells has been shown to undergo considerable down-regulation in cancer patients, which can significantly inhibit anti-tumor activity.

About FT596FT596 is an investigational, universal, off-the-shelf natural killer (NK) cell cancer immunotherapy derived from a clonal master induced pluripotent stem cell (iPSC) line engineered with three anti-tumor functional modalities: a proprietary chimeric antigen receptor (CAR) optimized for NK cell biology, which contains a NKG2D transmembrane domain, a 2B4 co-stimulatory domain and a CD3-zeta signaling domain, that targets B-cell antigen CD19; a novel high-affinity 158V, non-cleavable CD16 Fc receptor that has been modified to augment antibody-dependent cellular cytotoxicity by preventing CD16 down-regulation and enhancing CD16 binding to tumor-targeting antibodies; and an IL-15 receptor fusion (IL-15RF) that promotes enhanced NK cell activity. The FDA has allowed investigation of FT596 in an open-label Phase 1 clinical trial as a monotherapy, in combination with rituximab for the treatment of advanced B-cell lymphoma, and in combination with obinutuzumab for the treatment of chronic lymphocytic leukemia. In preclinical studies of FT596, the Company has demonstrated that dual activation of the CAR19 and CD16 receptors, in combination with IL-15RF signaling, convey synergistic anti-tumor activity. Increased degranulation and cytokine release were observed upon dual receptor activation in lymphoma cancer cells as compared to activation of each receptor alone, indicating that multi-antigen engagement may elicit a deeper and more durable response. Additionally, in a mixed cellular composition cytotoxicity assay comprised of CD19+ and CD19- tumor cells, FT596 combined with CD20-directed monoclonal antibody therapy effectively eliminated the heterogeneous population of tumor cells, a result that was not observed with single-antigen targeted CAR19 T cells.

About Fate Therapeutics, Inc.Fate Therapeutics is a clinical-stage biopharmaceutical company dedicated to the development of first-in-class cellular immunotherapies for cancer and immune disorders. The Company has established a leadership position in the clinical development and manufacture of universal, off-the-shelf cell products using its proprietary induced pluripotent stem cell (iPSC) product platform. The Companys immuno-oncology product candidates include natural killer (NK) cell and T-cell cancer immunotherapies, which are designed to synergize with well-established cancer therapies, including immune checkpoint inhibitors and monoclonal antibodies, and to target tumor-associated antigens with chimeric antigen receptors (CARs). The Companys immuno-regulatory product candidates include ProTmune, a pharmacologically modulated, donor cell graft that is currently being evaluated in a Phase 2 clinical trial for the prevention of graft-versus-host disease, and a myeloid-derived suppressor cell immunotherapy for promoting immune tolerance in patients with immune disorders. Fate Therapeutics is headquartered in San Diego, CA. For more information, please visit http://www.fatetherapeutics.com.

Forward-Looking StatementsThis release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 including statements regarding the Companys results of operations, financial condition and sufficiency of its cash and cash equivalents to fund its operations, as well as statements regarding the advancement of and plans related to its product candidates, clinical studies and preclinical research and development programs, the Companys progress, plans and timelines for the manufacture and clinical investigation of its product candidates, the timing for the Companys receipt of data from its clinical trials and preclinical studies, the Companys development and regulatory strategy, and the therapeutic and market potential of the Companys product candidates. These and any other forward-looking statements in this release are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that results observed in prior studies of the Companys product candidates, including preclinical studies and clinical trials, will not be observed in ongoing or future studies involving these product candidates, the risk of a delay or difficulties in the manufacturing of the Companys product candidates or in the initiation of, or enrollment of subjects in, any clinical studies, the risk that the Company may cease or delay preclinical or clinical development of any of its product candidates for a variety of reasons (including requirements that may be imposed by regulatory authorities on the initiation or conduct of clinical trials or to support regulatory approval, difficulties or delays in subject enrollment in current and planned clinical trials, difficulties in manufacturing or supplying the Companys product candidates for clinical testing, and any adverse events or other negative results that may be observed during preclinical or clinical development), and the risk that the Companys expenditures may exceed current expectations for a variety of reasons. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Companys actual results to differ from those contained in the forward-looking statements, see the risks and uncertainties detailed in the Companys periodic filings with the Securities and Exchange Commission, including but not limited to the Companys most recently filed periodic report, and from time to time in the Companys press releases and other investor communications.Fate Therapeutics is providing the information in this release as of this date and does not undertake any obligation to update any forward-looking statements contained in this release as a result of new information, future events or otherwise.

Availability of Other Information about Fate Therapeutics, Inc.Investors and others should note that the Company routinely communicates with investors and the public using its website (www.fatetherapeutics.com) and its investor relations website (ir.fatetherapeutics.com) including, without limitation, through the posting of investor presentations, SEC filings, press releases, public conference calls and webcasts on these websites. The information posted on these websites could be deemed to be material information. As a result, investors, the media, and others interested in Fate Therapeutics are encouraged to review this information on a regular basis. The contents of the Companys website, or any other website that may be accessed from the Companys website, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended.

Condensed Consolidated Statements of Operations and Comprehensive Loss(in thousands, except share and per share data)(unaudited)

Condensed Consolidated Balance Sheets(in thousands)(unaudited)

Contact:Christina TartagliaStern Investor Relations, Inc.212.362.1200christina@sternir.com

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Fate Therapeutics Reports Third Quarter 2019 Financial Results and Highlights Operational Progress - Yahoo Finance

MD Anderson and Takeda Announce Collaboration to Accelerate the Development of Clinical-Stage, Off-The-Shelf CAR NK-Cell Therapy Platform – Newswise

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Newswise HOUSTON and OSAKA, JAPAN --The University of Texas MD Anderson Cancer Center and Takeda Pharmaceutical Company Limited today announced an exclusive license agreement and research agreement to develop cord blood-derived chimeric antigen receptor-directed natural killer (CAR NK)-cell therapies, armored with IL-15, for the treatment of B-cell malignancies and other cancers.

Under the agreement, Takeda will receive access to MD Andersons CAR NK platform and the exclusive rights to develop and commercialize up to four programs, including a CD19-targeted CAR NK-cell therapy and a B-cell maturation antigen (BCMA)-targeted CAR NK-cell therapy. Takeda and MD Anderson also will conduct a research collaboration to further develop these CAR NK programs.

Our vision is to improve upon existing treatments by developing armored CAR NKs that could be administered off-the-shelf in an outpatient settingenabling more patients to be treated effectively, quickly and with minimal toxicities, said Katy Rezvani, M.D., Ph.D., professor of Stem Cell Transplantation and Cellular Therapy at MD Anderson. With their expertise in hematologic malignancies and commitment to developing next-generation cell therapies, Takeda is the ideal collaborator to help our team advance CAR NK-cell therapies to patients in need of treatments.

A Novel Approach to Delivering Off-the-Shelf CARs in an Outpatient Setting

MD Andersons allogeneic CAR NK platform isolates NK cells from umbilical cord blood and engineers them to express CARs against specified cancer targets. CAR NK cells are modified with a retroviral vector to deliver genes and enhance their effectiveness to attack specific tumors. A CD19 CAR increases the cells specificity for B-cell malignancies while the immunocytokine IL-15 enhances the proliferation and survival of the CAR NK cells in the body.

In contrast to current CAR T-cell therapies that utilize a patients own genetically modified T-cells and require a multi-week manufacturing process, CAR NK cells are intended to be manufactured from a non-related donor source and stored for off-the-shelf use, allowing treatment to be delivered more rapidly.

It is anticipated that the CD19 CAR NK-cell therapy could be administered in an outpatient setting. In an ongoing phase 1/2a clinical study treating patients with relapsed and refractory B-cell malignances, the CD19 CAR NK-cell therapy has not been associated with the severe cytokine release syndrome (CRS) or neurotoxicity observed with existing CAR-T therapies.

The development of MD Andersons CAR NK platform is led by Dr. Rezvani and is further supported by the adoptive cell therapy platform, Chronic Lymphocytic Leukemia Moon Shot and B-Cell Lymphoma Moon Shot, all part of the institutions Moon Shots Program, a collaborative effort to rapidly develop scientific discoveries into meaningful clinical advances that save patients lives.

Takeda: Accelerating the Development of Multiple Next-Generation CAR Platforms

MD Andersons CAR NK platform represents the curative potential of cell therapies, which is why we are establishing the CD19 CAR NK as our lead cell therapy candidate in oncology, said Andy Plump, M.D., Ph.D., President of Research and Development at Takeda. We need to work swiftly and with purpose, and as such, we intend to initiate a pivotal study of the CD19 CAR NK in 2021.

In addition to CAR NK-cell therapies, Takeda and its partners are investigating multiple approaches to improving the safety, efficacy and accessibility of first-generation CAR T-cell therapies including gamma delta CAR Ts, induced pluripotent stem cell-derived CAR Ts, CAR Ts targeting solid tumors, and other next-generation approaches. Takeda plans to advance five oncology cell therapies to the clinic by the end of FY20. These platforms are being developed both with partners and by applying the expertise of Takedas translational cell therapy engine which provides bioengineering, chemistry, manufacturing and control (CMC), clinical and translational capabilities in a single footprint to overcome many of the manufacturing challenges experienced in cell therapy development.

Takeda is responsible for the development, manufacturing and commercialization of CAR NK products resulting under the agreement. MD Anderson will receive an upfront payment and is eligible to receive development and commercial milestones for each target as well as tiered royalties on net sales of any such CAR NK product.

MD Anderson and Takeda will continue research for the additional targets and CAR-NK platform under the direction of a joint research committee. MD Anderson will implement an Institutional Conflict of Interest Management and Monitoring Plan for this research.

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MD Anderson and Takeda Announce Collaboration to Accelerate the Development of Clinical-Stage, Off-The-Shelf CAR NK-Cell Therapy Platform - Newswise

Orchard Therapeutics Reports Third Quarter 2019 Financial Results and Achievement of Key 2019 Milestones for Neurometabolic and Primary Immune…

ASH Abstracts Published Today Confirm Achievement of All Key Endpoints in Wiskott-Aldrich Syndrome Registrational Trial and Demonstrate Consistency of Data Between the Fresh and Cryopreserved Formulations of Investigational Gene Therapies

Marketing Authorization Application in EU for Metachromatic Leukodystrophy on Track for Near-Term Submission Following Recent Presentation of Cryopreservation Data

Ended the Third Quarter of 2019 with Approximately $366.2M in Total Cash and Investments

Conference Call Scheduled for Today at 4:30 p.m. ET

BOSTON and LONDON, Nov. 06, 2019 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a leading commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies, today announced financial results and business highlights for the quarter ended September 30, 2019, as well as the achievement of certain corporate milestones for the primary immune deficiencies franchise.

We made important clinical and operational progress at Orchard over the past quarter, advancing our three lead programs toward regulatory submissions while moving additional programs through earlier stages of clinical development, said Mark Rothera, president and chief executive officer of Orchard Therapeutics. The data presented at several major medical conferences throughout the quarter showcase the profound impact that our hematopoietic stem cell-based gene therapies may have across multiple life-threatening diseases. We are working with care and urgency to make these therapies broadly available to patients who need them.

Recent Achievements for the Neurometabolic Franchise

Metachromatic Leukodystrophy (MLD):

Mucopolysaccharidosis type I (MPS-I):

Sanfilippo Syndrome type A (MPS-IIIA):

Upcoming Data Presentations for Primary Immune Deficiencies Franchise

Third Quarter 2019 Financial Results

Cash, cash equivalents and investments as of September 30, 2019, were $366.2 million compared to $335.9 million as of December 31, 2018. The increase was primarily driven by proceeds from the companys public equity offering in June 2019 and a credit facility entered in May 2019, partially offset by the net cash used to fund operations in the nine months ended September 30, 2019. During the three months ended September 30, 2019, the companys cash used to fund operations included the paydown of accrued liabilities as of June 30, 2019, for the upfront payment and a milestone payment related to the license of a clinical-stage MPS-I program, or OTL-203.

During the three months ended September 30, 2019, the company recognized $1.9 million in revenue related to European sales of Strimvelis, the first gene therapy approved by the European Medicines Agency for ADA-SCID.

Research and development expenses were $28.5 million for the three months ended September 30, 2019 compared to $27.7 million in the same period in 2018. R&D expenses include the costs of clinical trials and preclinical work on the companys portfolio of investigational gene therapies, as well as costs related to regulatory, manufacturing, license fees and milestone payments under the companys agreements with third parties, and personnel costs to support these activities. The company expects R&D expenses to continue to increase as its programs advance through development.

Selling, general and administrative expenses were $14.2 million for the three months ended September 30, 2019, compared to $7.5 million in the same period in 2018. The increase was primarily due to increased investment to prepare for the potential commercialization of multiple late-stage programs, as well as higher costs to support public company operations.

Net loss attributable to ordinary shareholders was $36.7 million for the three months ended September 30, 2019, compared to $33.9 million in the same period in 2018. The increase in net loss as compared to the prior year was primarily due to higher costs related to pre-launch activities on the companys later-stage programs in development and expenses associated with being a public company, partially offset by higher interest income. The company had 97.8 million weighted average ordinary shares outstanding for the three months ended September 30, 2019.

The company expects that its existing cash, cash equivalents and investments will enable funding of its anticipated operating and capital expenditure requirements into the second half of 2021.

Orchard is well positioned to enter its next phase of growth as a company with potentially multiple commercial-stage gene therapies, building on a proven track record in identifying and treating patients with Strimvelis, the first ex vivo HSC gene therapy on the market, said Frank Thomas, Orchards chief financial officer and chief business officer. Having treated multiple patients with Strimvelis this quarter at a single center in Milan, Italy, we have deepened our understanding of the support required for patients and families along their treatment journey, successfully secured reimbursement for cross-border healthcare delivery within Europe and completed the manufacturing and release process scheduled and tailored for each individual patient. We look forward to expanding these capabilities and applying key learnings to future commercial gene therapies at multiple centers around the world using cryopreserved formulations of our future products, if approved.

Conference Call & Webcast Information

Orchard will host a conference call and live webcast with slides today at 4:30 p.m. ET to discuss the third quarter results and recent and upcoming business activities. To participate in the conference call, please dial 1-866-987-6504 (domestic) or +1-602-563-8620 (international) and refer to conference ID 8413109. A live webcast of the presentation will be available under "News & Events" in the Investors & Media section of the company's website at orchard-tx.com and a replay will be archived on the Orchard website following the presentation.

About Orchard

Orchard Therapeutics is a fully integrated commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies.

Orchards portfolio of ex vivo, autologous, hematopoietic stem cell (HSC) based gene therapies includes Strimvelis, a gammaretroviral vector-based gene therapy and the first such treatment approved by the European Medicines Agency for severe combined immune deficiency due to adenosine deaminase deficiency (ADA-SCID). Additional programs for neurometabolic disorders, primary immune deficiencies and hemoglobinopathies are all based on lentiviral vector-based gene modification of autologous HSCs and include three advanced registrational studies for metachromatic leukodystrophy (MLD), ADA-SCID and Wiskott-Aldrich syndrome (WAS), clinical programs for X-linked chronic granulomatous disease (X-CGD), transfusion-dependent beta-thalassemia (TDT) and mucopolysaccharidosis type I (MPS-I), as well as an extensive preclinical pipeline. Strimvelis, as well as the programs in MLD, WAS and TDT were acquired by Orchard from GSK in April 2018 and originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy, initiated in 2010.

Orchard currently has offices in the U.K. and the U.S., including London, San Francisco and Boston.

Forward-Looking Statements

This press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements may be identified by words such as anticipates, believes, expects, intends, projects, and future or similar expressions that are intended to identify forward-looking statements. Forward-looking statements include express or implied statements relating to, among other things, the therapeutic potential of Orchards product candidates, including the product candidate or candidates referred to in this release, Orchards expectations regarding the timing of regulatory submissions for approval of its product candidates, including the product candidate or candidates referred to in this release, the timing of interactions with regulators and regulatory submissions related to ongoing and new clinical trials for its product candidates, the timing of announcement of clinical data for its product candidates and the likelihood that such data will be positive and support further clinical development and regulatory approval of these product candidates, the likelihood of approval of such product candidates by the applicable regulatory authorities, and the companys financial condition and cash runway into the second half of 2021. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, the risks and uncertainties include, without limitation: the risk that any one or more of Orchards product candidates, including the product candidate or candidates referred to in this release, will not be successfully developed or commercialized, the risk of cessation or delay of any of Orchards ongoing or planned clinical trials, the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates,the delay of any of Orchards regulatory submissions, the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates, the receipt of restricted marketing approvals, and the risk of delays in Orchards ability to commercialize its product candidates, if approved. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards annual report on Form 20-F for the year ended December 31, 2018, as filed with the U.S. Securities and Exchange Commission (SEC) on March 22, 2019, as well as subsequent filings and reports filed with the SEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Condensed Consolidated Statements of Operations(In thousands, except share and per share amounts) (unaudited)

Condensed Consolidated Balance Sheets(In thousands) (unaudited)

Contact:Renee Leck, Director of Investor Relations Orchard Therapeutics+1 862-242-0764Renee.Leck@orchard-tx.com

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Orchard Therapeutics Reports Third Quarter 2019 Financial Results and Achievement of Key 2019 Milestones for Neurometabolic and Primary Immune...

Celgene to Present New and Updated Data on Key Hematology Pipeline Therapies at American Society of Hematology (ASH) 2019 Annual Meeting – Business…

SUMMIT, N.J.--(BUSINESS WIRE)--Celgene Corporation (NASDAQ: CELG) today announced data from nearly 70 Company-sponsored, global alliance and investigator-initiated clinical studies evaluating Celgenes investigational and approved therapies will be presented at the 61st American Society of Hematology (ASH) Annual Meeting & Exposition, December 7-10, in Orlando, Fla.

Celgene has a deep and ongoing commitment to innovative research and development in treatments for serious blood disorders, with the potential to transform patient outcomes, said Alise Reicin, M.D., President, Global Clinical Development for Celgene. We look forward to ASH as an opportunity to highlight our commitment and leadership in the research and development of novel therapies for the treatment of blood cancers through new insights in both CD19 and BCMA targeted cell therapies and important progress in our myeloid pipeline.

In leukemia and lymphoma, highlighted studies this year include safety and efficacy results from the pivotal TRANSCEND NHL-001 study of an investigational CD-19 targeted chimeric antigen receptor (CAR) T cell therapy lisocabtagene maraleucel (liso-cel) in relapsed/refractory large B-cell non-Hodgkin lymphoma. Additional liso-cel data from three ongoing studies will evaluate the use of the therapy in an outpatient setting, as well as in transplant noneligible patients with relapsed/refractory large B-cell non-Hodgkin lymphoma (PILOT) and in patients with relapsed/refractory chronic lymphocytic leukemia (TRANSCEND CLL-004).

In multiple myeloma, other notable investigational cell therapy abstracts include the first phase 1 clinical data from the bi-specific T-Cell Engager (TCE) CC-93269 and updated phase 1 clinical data from CAR T program, bb21217, both targeting the B-cell maturation antigen (BCMA) in relapsed/refractory disease.

Several abstracts focusing on data in myeloid diseases including longer-term response data from the phase 3 MEDALIST study of luspatercept to treat anemia in patients with IPSS-R very low-, low-, or intermediate-risk myelodysplastic syndromes with ring sideroblasts who require red-blood-cell (RBC) transfusions will be presented. Additionally, the first data from a phase 2 study of luspatercept in myelofibrosis-associated anemia, results from a study of fedratinib in myelofibrosis patients with low platelet counts, and the first data from CELMoD agent CC-90009, a GSPT1 degrader in relapsed or refractory acute myeloid leukemia (AML) will be presented.

Selected abstracts include*:

Lymphoma & Chronic Lymphocytic Leukemia

Multiple Myeloma

Myeloid Diseases

Beta thalassemia

A complete listing of abstracts can be found at https://www.hematology.org/Annual-Meeting/abstracts/

The safety and efficacy of investigational agents and/or investigational uses of approved marketed products have not been established. There is no guarantee that the agents will receive health authority approval or become commercially available in any country for the uses being investigated.

*All times Eastern Time

About REVLIMID

REVLIMID (lenalidomide) in combination with dexamethasone (dex) is indicated for the treatment of adult patients with multiple myeloma (MM)

REVLIMID is indicated as maintenance therapy in adult patients with MM following autologous hematopoietic stem cell transplantation (auto-HSCT)

REVLIMID is indicated for the treatment of adult patients with transfusion-dependent anemia due to low-or intermediate-1risk myelodysplastic syndromes (MDS) associated with a deletion 5q cytogenetic abnormality with or without additional cytogenetic abnormalities

REVLIMID is indicated for the treatment of adult patients with mantle cell lymphoma (MCL) whose disease has relapsed or progressed after two prior therapies, one of which included bortezomib

REVLIMID in combination with a rituximab product is indicated for the treatment of adult patients with previously treated follicular lymphoma (FL)

REVLIMID in combination with a rituximab product is indicated for the treatment of adult patients with previously treated marginal zone lymphoma (MZL)

REVLIMID is not indicated and is not recommended for the treatment of patients with chronic lymphocytic leukemia (CLL) outside of controlled clinical trials

REVLIMID is only available through a restricted distribution program, REVLIMID REMS.

Important Safety Information

WARNING: EMBRYO-FETAL TOXICITY, HEMATOLOGIC TOXICITY, and VENOUS and ARTERIAL THROMBOEMBOLISM

Embryo-Fetal Toxicity

Do not use REVLIMID during pregnancy. Lenalidomide, a thalidomide analogue, caused limb abnormalities in a developmental monkey study. Thalidomide is a known human teratogen that causes severe life-threatening human birth defects. If lenalidomide is used during pregnancy, it may cause birth defects or embryo-fetal death. In females of reproductive potential, obtain 2 negative pregnancy tests before starting REVLIMID treatment. Females of reproductive potential must use 2 forms of contraception or continuously abstain from heterosexual sex during and for 4 weeks after REVLIMID treatment. To avoid embryo-fetal exposure to lenalidomide, REVLIMID is only available through a restricted distribution program, the REVLIMID REMS program.

Information about the REVLIMID REMS program is available at http://www.celgeneriskmanagement.com or by calling the manufacturers toll-free number 1-888-423-5436.

Hematologic Toxicity (Neutropenia and Thrombocytopenia)

REVLIMID can cause significant neutropenia and thrombocytopenia. Eighty percent of patients with del 5q MDS had to have a dose delay/reduction during the major study. Thirty-four percent of patients had to have a second dose delay/reduction. Grade 3 or 4 hematologic toxicity was seen in 80% of patients enrolled in the study. Patients on therapy for del 5q MDS should have their complete blood counts monitored weekly for the first 8 weeks of therapy and at least monthly thereafter. Patients may require dose interruption and/or reduction. Patients may require use of blood product support and/or growth factors.

Venous and Arterial Thromboembolism

REVLIMID has demonstrated a significantly increased risk of deep vein thrombosis (DVT) and pulmonary embolism (PE), as well as risk of myocardial infarction and stroke in patients with MM who were treated with REVLIMID and dexamethasone therapy. Monitor for and advise patients about signs and symptoms of thromboembolism. Advise patients to seek immediate medical care if they develop symptoms such as shortness of breath, chest pain, or arm or leg swelling. Thromboprophylaxis is recommended and the choice of regimen should be based on an assessment of the patients underlying risks.

CONTRAINDICATIONS

Pregnancy: REVLIMID can cause fetal harm when administered to a pregnant female and is contraindicated in females who are pregnant. If this drug is used during pregnancy or if the patient becomes pregnant while taking this drug, the patient should be apprised of the potential risk to the fetus

Severe Hypersensitivity Reactions: REVLIMID is contraindicated in patients who have demonstrated severe hypersensitivity (e.g., angioedema, Stevens-Johnson syndrome, toxic epidermal necrolysis) to lenalidomide

WARNINGS AND PRECAUTIONS

Embryo-Fetal Toxicity: See Boxed WARNINGS

REVLIMID REMS Program: See Boxed WARNINGS: Prescribers and pharmacies must be certified with the REVLIMID REMS program by enrolling and complying with the REMS requirements; pharmacies must only dispense to patients who are authorized to receive REVLIMID. Patients must sign a Patient-Physician Agreement Form and comply with REMS requirements; female patients of reproductive potential who are not pregnant must comply with the pregnancy testing and contraception requirements and males must comply with contraception requirements

Hematologic Toxicity: REVLIMID can cause significant neutropenia and thrombocytopenia. Monitor patients with neutropenia for signs of infection. Advise patients to observe for bleeding or bruising, especially with use of concomitant medications that may increase risk of bleeding. Patients may require dose interruption and/or dose reduction. MM: Monitor complete blood counts (CBC) in patients taking REVLIMID + dexamethasone or REVLIMID as maintenance therapy, every 7 days for the first 2 cycles, on days 1 and 15 of cycle 3, and every 28 days thereafter. MDS: Monitor CBC in patients on therapy for del 5q MDS, weekly for the first 8 weeks of therapy and at least monthly thereafter. See Boxed WARNINGS for further information. MCL: Monitor CBC in patients taking REVLIMID for MCL weekly for the first cycle (28 days), every 2 weeks during cycles 2-4, and then monthly thereafter. FL/MZL: Monitor CBC in patients taking REVLIMID for FL or MZL weekly for the first 3 weeks of Cycle 1 (28 days), every 2 weeks during Cycles 2-4, and then monthly thereafter

Venous and Arterial Thromboembolism: See Boxed WARNINGS: Venous thromboembolic events (DVT and PE) and arterial thromboses (MI and CVA) are increased in patients treated with REVLIMID. Patients with known risk factors, including prior thrombosis, may be at greater risk and actions should be taken to try to minimize all modifiable factors (e.g., hyperlipidemia, hypertension, smoking). Thromboprophylaxis is recommended and the regimen should be based on the patients underlying risks. ESAs and estrogens may further increase the risk of thrombosis and their use should be based on a benefit-risk decision

Increased Mortality in Patients With CLL: In a clinical trial in the first-line treatment of patients with CLL, single-agent REVLIMID therapy increased the risk of death as compared to single-agent chlorambucil. Serious adverse cardiovascular reactions, including atrial fibrillation, myocardial infarction, and cardiac failure, occurred more frequently in the REVLIMID arm. REVLIMID is not indicated and not recommended for use in CLL outside of controlled clinical trials

Second Primary Malignancies (SPM): In clinical trials in patients with MM receiving REVLIMID and in patients with FL or MZL receiving REVLIMID + rituximab therapy, an increase of hematologic plus solid tumor SPM, notably AML, have been observed. In MM patients, MDS was also observed. Monitor patients for the development of SPM. Take into account both the potential benefit of REVLIMID and risk of SPM when considering treatment

Increased Mortality with Pembrolizumab: In clinical trials in patients with MM, the addition of pembrolizumab to a thalidomide analogue plus dexamethasone resulted in increased mortality. Treatment of patients with MM with a PD-1-or PD-L1- blocking antibody in combination with a thalidomide analogue plus dexamethasone is not recommended outside of controlled clinical trials

Hepatotoxicity: Hepatic failure, including fatal cases, has occurred in patients treated with REVLIMID + dexamethasone. Pre-existing viral liver disease, elevated baseline liver enzymes, and concomitant medications may be risk factors. Monitor liver enzymes periodically. Stop REVLIMID upon elevation of liver enzymes. After return to baseline values, treatment at a lower dose may be considered

Severe Cutaneous Reactions Including Hypersensitivity Reactions: Angioedema and severe cutaneous reactions including Stevens-Johnson syndrome (SJS), toxic epidermal necrolysis (TEN), and drug reaction with eosinophilia and systemic symptoms (DRESS) have been reported. These events can be fatal. Patients with a prior history of Grade 4 rash associated with thalidomide treatment should not receive REVLIMID. REVLIMID interruption or discontinuation should be considered for Grade 2-3 skin rash. REVLIMID must be discontinued for angioedema, Grade 4 rash, exfoliative or bullous rash, or if SJS, TEN, or DRESS is suspected and should not be resumed following discontinuation for these reactions

Tumor Lysis Syndrome (TLS): Fatal instances of TLS have been reported during treatment with REVLIMID. The patients at risk of TLS are those with high tumor burden prior to treatment. Closely monitor patients at risk and take appropriate preventive approaches

Tumor Flare Reaction (TFR): TFR has occurred during investigational use of REVLIMID for CLL and lymphoma. Monitoring and evaluation for TFR is recommended in patients with MCL, FL, or MZL. Tumor flare may mimic the progression of disease (PD). In patients with Grade 3 or 4 TFR, it is recommended to withhold treatment with REVLIMID until TFR resolves to Grade 1. REVLIMID may be continued in patients with Grade 1 and 2 TFR without interruption or modification, at the physicians discretion

Impaired Stem Cell Mobilization: A decrease in the number of CD34+ cells collected after treatment (>4 cycles) with REVLIMID has been reported. Consider early referral to transplant center to optimize timing of the stem cell collection

Thyroid Disorders: Both hypothyroidism and hyperthyroidism have been reported. Measure thyroid function before starting REVLIMID treatment and during therapy

Early Mortality in Patients With MCL: In another MCL study, there was an increase in early deaths (within 20 weeks); 12.9% in the REVLIMID arm versus 7.1% in the control arm. Risk factors for early deaths include high tumor burden, MIPI score at diagnosis, and high WBC at baseline (10 x 109/L)

ADVERSE REACTIONS

Multiple Myeloma

Myelodysplastic Syndromes

Mantle Cell Lymphoma

Follicular Lymphoma/Marginal Zone Lymphoma

DRUG INTERACTIONS

Periodically monitor digoxin plasma levels due to increased Cmax and AUC with concomitant REVLIMID therapy. Patients taking concomitant therapies such as erythropoietin-stimulating agents or estrogen-containing therapies may have an increased risk of thrombosis. It is not known whether there is an interaction between dexamethasone and warfarin. Close monitoring of PT and INR is recommended in patients with MM taking concomitant warfarin

USE IN SPECIFIC POPULATIONS

Please see full Prescribing Information, including Boxed WARNINGS, for REVLIMID.

About INREBIC

INREBIC (fedratinib) is indicated for the treatment of adult patients with intermediate-2 or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis (MF).

IMPORTANT SAFETY INFORMATION

WARNING: ENCEPHALOPATHY INCLUDING WERNICKES

Serious and fatal encephalopathy, including Wernickes, has occurred in patients treated with INREBIC. Wernickes encephalopathy is a neurologic emergency. Assess thiamine levels in all patients prior to starting INREBIC, periodically during treatment, and as clinically indicated. Do not start INREBIC in patients with thiamine deficiency; replete thiamine prior to treatment initiation. If encephalopathy is suspected, immediately discontinue INREBIC and initiate parenteral thiamine. Monitor until symptoms resolve or improve and thiamine levels normalize.

WARNINGS AND PRECAUTIONS

Encephalopathy, including Wernickes: Serious and fatal encephalopathy, including Wernickes encephalopathy, has occurred in INREBIC-treated patients. Serious cases were reported in 1.3% (8/608) of patients treated with INREBIC in clinical trials and 0.16% (1/608) of cases were fatal.

Wernickes encephalopathy is a neurologic emergency resulting from thiamine (Vitamin B1) deficiency. Signs and symptoms of Wernickes encephalopathy may include ataxia, mental status changes, and ophthalmoplegia (e.g., nystagmus, diplopia). Any change in mental status, confusion, or memory impairment should raise concern for potential encephalopathy, including Wernickes, and prompt a full evaluation including a neurologic examination, assessment of thiamine levels, and imaging. Assess thiamine levels in all patients prior to starting INREBIC, periodically during treatment, and as clinically indicated. Do not start INREBIC in patients with thiamine deficiency; replete thiamine prior to treatment initiation. If encephalopathy is suspected, immediately discontinue INREBIC and initiate parenteral thiamine. Monitor until symptoms resolve or improve and thiamine levels normalize.

Anemia: New or worsening Grade 3 anemia occurred in 34% of INREBIC-treated patients. The median time to onset of the first Grade 3 anemia was approximately 2 months, with 75% of cases occurring within 3 months. Mean hemoglobin levels reached nadir after 12 to 16 weeks with partial recovery and stabilization after 16 weeks. Red blood cell transfusions were received by 51% of INREBIC-treated patients and permanent discontinuation of INREBIC occurred due to anemia in 1% of patients. Consider dose reduction for patients who become red blood cell transfusion dependent

Thrombocytopenia: New or worsening Grade 3 thrombocytopenia during the randomized treatment period occurred in 12% of INREBIC-treated patients. The median time to onset of the first Grade 3 thrombocytopenia was approximately 1 month; with 75% of cases occurring within 4 months. Platelet transfusions were received by 3.1% INREBIC-treated patients. Permanent discontinuation of treatment due to thrombocytopenia and bleeding that required clinical intervention both occurred in 2.1% of INREBIC-treated patients. Obtain a complete blood count (CBC) at baseline, periodically during treatment, and as clinically indicated. For Grade 3 thrombocytopenia with active bleeding or Grade 4 thrombocytopenia, interrupt INREBIC until resolved to less than or equal to Grade 2 or baseline. Restart dose at 100 mg daily below the last given dose and monitor platelets as clinically indicated.

Gastrointestinal Toxicity: Gastrointestinal toxicities are the most frequent adverse reactions in INREBIC-treated patients. During the randomized treatment period, diarrhea occurred in 66% of patients, nausea in 62% of patient and vomiting in 39% of patients. Grade 3 diarrhea 5% and vomiting 3.1% occurred. The median time to onset of any grade nausea, vomiting, and diarrhea was 1 day, with 75% of cases occurring within 2 weeks of treatment. Consider providing appropriate prophylactic anti-emetic therapy (e.g., 5-HT3 receptor antagonists) during INREBIC treatment. Treat diarrhea with anti-diarrheal medications promptly at the first onset of symptoms. Grade 3 or higher nausea, vomiting, or diarrhea not responsive to supportive measures within 48 hours, interrupt INREBIC until resolved to Grade 1 or less or baseline. Restart dose at 100 mg daily below the last given dose. Monitor thiamine levels and replete as needed.

Hepatic Toxicity: Elevations of ALT and AST (all grades) during the randomized treatment period occurred in 43% and 40%, respectively, with Grade 3 or 4 in 1% and 0%, respectively, of INREBIC-treated patients. The median time to onset of any grade transaminase elevation was approximately 1 month, with 75% of cases occurring within 3 months. Monitor hepatic function at baseline, periodically during treatment, and as clinically indicated. For Grade 3 or higher ALT and/or AST elevations (greater than 5 ULN), interrupt INREBIC dose until resolved to Grade 1 or less or to baseline. Restart dose at 100 mg daily below the last given dose. If re-occurrence of a Grade 3 or higher elevation of ALT/AST, discontinue treatment with INREBIC.

Amylase and Lipase Elevation: Grade 3 or higher amylase 2% and/or lipase 10% elevations developed in INREBIC-treated patients. The median time to onset of any grade amylase or lipase elevation was 15 days, with 75% of cases occurring within 1 month of starting treatment. One patient developed pancreatitis in the fedratinib clinical development program (n=608) and pancreatitis resolved with treatment discontinuation. Monitor amylase and lipase at baseline, periodically during treatment, and as clinically indicated. For Grade 3 or higher amylase and/or lipase elevations, interrupt INREBIC until resolved to Grade 1 or less or to baseline. Restart dose at 100 mg daily below the last given dose.

ADVERSE REACTIONS: The most common adverse reactions for INREBIC treated vs. placebo were diarrhea (66% vs. 16%), nausea (62% vs. 15%), anemia (40% vs. 14%), and vomiting (39% vs. 5%). Dosage interruptions due to an adverse reaction during the randomized treatment period occurred in 21% of patients who received INREBIC. Adverse reactions requiring dosage interruption in >3% of patients who received INREBIC included diarrhea and nausea. Dosage reductions due to an adverse reaction during the randomized treatment period occurred in 19% of patients who received INREBIC. Adverse reactions requiring dosage reduction in >2% of patients who received INREBIC included anemia (6%), diarrhea (3%), vomiting (3%), and thrombocytopenia (2%).

DRUG INTERACTIONS: Coadministration of INREBIC with a strong CYP3A4 inhibitor increases fedratinib exposure. Increased exposure may increase the risk of adverse reactions. Consider alternative therapies that do not strongly inhibit CYP3A4 activity. Alternatively, reduce the dose of INREBIC when administering with a strong CYP3A4 inhibitor. Avoid INREBIC with strong and moderate CYP3A4 inducers. Avoid INREBIC with dual CYP3A4 and CYP2C19 inhibitor. Coadministration of INREBIC with drugs that are CYP3A4 substrates, CYP2C19 substrates, or CYP2D6 substrates increases the concentrations of these drugs, which may increase the risk of adverse reactions of these drugs. Monitor for adverse reactions and adjust the dose of drugs that are CYP3A4, CYP2C19, or CYP2D6 substrates as necessary when coadministered with INREBIC.

PREGNANCY/LACTATION: Consider the benefits and risks of INREBIC for the mother and possible risks to the fetus when prescribing INREBIC to a pregnant woman. Due to the potential for serious adverse reactions in a breastfed child, advise patients not to breastfeed during treatment with INREBIC, and for at least 1 month after the last dose.

RENAL IMPAIRMENT: Reduce INREBIC dose when administered to patients with severe renal impairment. No modification of the starting dose is recommended for patients with mild to moderate renal impairment. Due to potential increase of exposure, patients with preexisting moderate renal impairment require more intensive safety monitoring, and if necessary, dose modifications based on adverse reactions.

HEPATIC IMPAIRMENT: Avoid use of INREBIC in patients with severe hepatic impairment.

Please see full Prescribing Information, including Boxed WARNING.

About Celgene

Celgene Corporation, headquartered in Summit, New Jersey, is an integrated global biopharmaceutical company engaged primarily in the discovery, development and commercialization of innovative therapies for the treatment of cancer and inflammatory diseases through next-generation solutions in protein homeostasis, immuno-oncology, epigenetics, immunology and neuro-inflammation. For more information, please visit http://www.celgene.com. Follow Celgene on Social Media: Twitter, Pinterest, LinkedIn, Facebook and YouTube.

FORWARD-LOOKING STATEMENTS

This press release contains forward-looking statements, which are generally statements that are not historical facts. Forward-looking statements can be identified by the words "expects," "anticipates," "believes," "intends," "estimates," "plans," "will," "outlook" and similar expressions. Forward-looking statements are based on management's current plans, estimates, assumptions and projections, and speak only as of the date they are made. We undertake no obligation to update any forward-looking statement in light of new information or future events, except as otherwise required by law. Forward-looking statements involve inherent risks and uncertainties, most of which are difficult to predict and are generally beyond our control. Actual results or outcomes may differ materially from those implied by the forward-looking statements as a result of the impact of a number of factors, many of which are discussed in more detail in our Annual Report on Form 10-K and our other reports filed with the U.S. Securities and Exchange Commission, including factors related to the proposed transaction between Bristol-Myers Squibb and Celgene, such as, but not limited to, the risks that: management's time and attention is diverted on transaction related issues, including the planned divestiture of OTEZLA; disruption from the proposed transaction makes it more difficult to maintain business, contractual and operational relationships; legal proceedings are instituted against Bristol-Myers Squibb, Celgene or the combined company; and Bristol-Myers Squibb, Celgene or the combined company is unable to retain key personnel.

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Originally posted here:
Celgene to Present New and Updated Data on Key Hematology Pipeline Therapies at American Society of Hematology (ASH) 2019 Annual Meeting - Business...