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Cesca Therapeutics Forms Joint Venture with Healthbanks Biotech (USA) to Provide Immune Cell Banking and Cell Processing Services – P&T Community

By Stell Cell Research

RANCHO CORDOVA, Calif., Oct. 22, 2019 /PRNewswire/ -- Cesca Therapeutics Inc.(Nasdaq: KOOL), a market leader in automated cell processing and autologous cell therapies for regenerative medicine, and ThermoGenesis, its wholly owned device subsidiary, today announced that the company has entered into a definitive joint venture agreement with HealthBanks Biotech (USA) Inc., one of the world's leading stem cell bank networks, to commercialize its proprietary cell processing platform, CAR-TXpress, for use in immune cell banking as well as for cell-basedcontract development and manufacturing services (CMO/CDMO). The joint venture will be named ImmuneCyte Life Sciences Inc. ("ImmuneCyte") and is expected to officially launch during the fourth quarter of 2019.

Under terms of the agreement, ImmuneCyte will initially be owned 80% by HealthBanks Biotech and 20% by Cesca. Cesca will contribute to ImmuneCyte exclusive rights to use ThermoGenesis' proprietary cell processing technology for the immune cell banking business and non-exclusive rights for other cell-based contract development and manufacturing services. Cesca will also contribute its clinical development assets to the joint venture, as the company has decided to discontinue these activities in order to focus exclusively on the device business.

Once operational, ImmuneCyte will be among the first immune cell banks in the U.S. to provide clients with the opportunity to bank their own healthy immune cells for future use as a resource for cell-based immunotherapies, such as dendritic cell and chimeric antigen receptor (CAR) T-cell therapies. ImmuneCyte will utilize ThermoGenesis' proprietary CAR-TXpress platform which allows for the isolation of different components from 200 ml of blood in cGMP compliant, closed system. Given that the CAR-TXpress platform can increase cell processing efficiency by up to 16-fold as compared with the traditional, labor-intensive ficoll gradient centrifugation-based cell processing method, ImmuneCyte is expected to offer customers an unparalleled competitive advantage, including an ability to store their own immune cells at a tangibly lower cost.

"The ImmuneCyte joint venture will be paramount to the execution of our strategy to become a preferred cell processing and manufacturing solution provider in the cell and gene therapy field," said Dr. Chris Xu, Chairman and Chief Executive Officer of Cesca Therapeutics. "CAR-T therapeutic research is advancing rapidly. Partnering with HealthBanks Biotech, one of the foremost stem cell bank networks, with an experienced team and an established global infrastructure, will offer customers the ability to preserve younger, healthier and uncontaminated immune cells for potential future use. By applying our proprietary CAR-TXpress technology to immune cell banking and other CDMO cellular manufacturing services, we will allow for the manufacture and production of more effective and less costly immunotherapies."

In 2017, the U.S. Food and Drug Administration (FDA) approved two CAR-T cell therapies, under breakthrough designation, for the treatment of advanced B cell leukemia and lymphomas. Both use autologous (a patient's own) immune T cells to fight cancer and have reported an over 80% response rate in the "no-option" patient group, for those who have failed both chemo- and radiation therapies. This has helped to spur massive global interest for the development of additional CAR-T immunotherapies1. By the end of September 2019, there were over 800 CAR-T cell clinical trials registered on the http://www.clinicaltrials.gov website, targeting a wide variety of blood cancers and solid tumors.

Although highly effective, several recent studies on the eligibility of patients to enroll in CAR-T clinical trials showed that as many as 30-50% of cancer patients may not be eligible to enroll or to get sufficient CAR-T cells manufactured for the therapy. Reasons may include: (1) the function of the immune system declines with age and can be negatively affected by other medical conditions, (2) most standard cancer therapies, such as chemotherapy and radiation, destroy the immune system, and (3) in many cases of advanced cancer, cancer cells will enter circulation, invade and interfere with the body's natural production of immune cells. According to a recently reported JULIE trial, a CAR-T clinical trial in relapsed or refractory diffuse large B-cell lymphoma (DLBCL), one-third of the 238 screened patients failed to be enrolled, and more than half of the 238 failed to receive the intended CAR-T therapy2,3. ImmuneCyte will offer customers the ability to preserve younger, healthier and uncontaminated immune cells, for potential future use in advanced cancer immunotherapy.

About HealthBanks Biotech (USA) Inc.HealthBanks Biotech, headquartered in Irvine, CA, is one of the leading stem cell bank networks in the world and offers services globally through its sister companies located in the United States and other regions and nations. HealthBanks Biotech is accredited by the FDA, AABB, and CAP. The HealthBanks Biotech group was originally founded in 2001 with a vision that stem cells and cell and gene therapies could transform modern medicine. HealthBanks Biotech is a subsidiary of Boyalife Group, Inc. (USA), an affiliate of Boyalife (Hong Kong) Limited, the largest stockholder of Cesca. For more information about HealthBanks Biotech (USA) Inc., pleasevisit:www.healthbanks.us.

About ImmuneCyte Life Sciences Inc.ImmuneCyte will provide clients with the opportunity to bank their own immune cells when the cells are "healthy and unaffected" as a future resource for cellular immunotherapies, such as CAR-T. ImmuneCyte utilizes a proprietary CAR-TXpress platform, a GMP compliant close-system capable of automated separating and cryopreserving different components from blood.For more information about ImmuneCyte Life Sciences Inc., pleasevisit:www.immunecyte.com.

About Cesca Therapeutics Inc.Cesca Therapeuticsdevelops, commercializes and markets a range of automated technologies for CAR-T and other cell-based therapies. Its device division, ThermoGenesis develops, commercializes and markets a full suite of solutions for automated clinical biobanking, point-of-care applications, and automation for immuno-oncology. The Company has developed a semi- automated, functionally closed CAR-TXpressplatform to streamline the manufacturing process for the emerging CAR-T immunotherapy market. For more information about Cesca and ThermoGenesis, pleasevisit: http://www.cescatherapeutics.com.

Company Contact:Wendy Samford916-858-5191ir@thermogenesis.com

Investor Contact:Paula Schwartz,Rx Communications917-322-2216pschwartz@rxir.com

References:

1. Facts About Chimeric Antigen Receptor (CAR) T-Cell Therapy, Leukemia and Lymphoma Society (2018). https://www.lls.org

2. Updated Analysis of JULIET Trial: Tisagenlecleucel in Relapsed or Refractory DLBCL (2018).

3. Eligibility Criteria for CAR-T Trials and Survival Rates in Chemorefractory DLBCL. Journal of Clinical Pathways (2018).

View original content:http://www.prnewswire.com/news-releases/cesca-therapeutics-forms-joint-venture-with-healthbanks-biotech-usa-to-provide-immune-cell-banking-and-cell-processing-services-300942618.html

SOURCE Cesca Therapeutics Inc.

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Cesca Therapeutics Forms Joint Venture with Healthbanks Biotech (USA) to Provide Immune Cell Banking and Cell Processing Services - P&T Community

CST and Cell Press launch Cell Mentor in China – BSA bureau

By Stell Cell Research

Online Resource to Help Scientist Achieve Experimental and Career Success Now Available

US headquartered Cell Signaling Technology(CST), a leading provider of antibodies, kits, and services, andCell Press, a leading publisher of over 40 primary research and review journals, have announced thatCell Mentor, an educational resource for biology students and researchers is available inChina.

Originally launched at AACR onMarch 30th, Cell Mentor enables biology students and researchers to easily navigate their careers, get published, and strengthen their laboratory skills to reach experimental success. Scientists inChinawill now have the ability to utilize Cell Mentor in their native Mandarin language atwww.cellmentor.cn.

Cell Mentor is full of educational content from both Cell Press and CST and information is presented according to the types of challenges students and researchers face during their careers. It is designed to address real-life quandaries faced by students and researchers in and out of the lab.

To celebrate the new Cell Mentor section, CST hosted IUIS 2019 Featured Forum: New Frontiers in Immunology, New Beginnings with Cell Mentor onOctober 21st. In the forum, renowned opinion leaders in immunology introduced the new frontiers in infection andimmunity, NK cell-based immunotherapy of cancer,stem cell research and applications.

In addition,Jay Dong, Vice President and General ManagerChinaandAsia Pacificat CST introduced the origin of Cell Mentor and how it can facilitate scientific experiments, andPeter Lee, Editor-In-Chief of Immunity and Publishing Director at Cell Press, discussed how to publish papers inCell,Immunityand beyond.

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CST and Cell Press launch Cell Mentor in China - BSA bureau

How stem cells are helping to mend broken hearts – Belfast Telegraph

By Stell Cell Research

How stem cells are helping to mend broken hearts

BelfastTelegraph.co.uk

In recent years, it's become apparent that stem cells will be a key ingredient of future medical treatments. And nowhere is the use of these 'building block cells' more important than through their amazing ability to repair the heart.

https://www.belfasttelegraph.co.uk/life/features/how-stem-cells-are-helping-to-mend-broken-hearts-38616582.html

https://www.belfasttelegraph.co.uk/life/features/article38616581.ece/72ec8/AUTOCROP/h342/2019-10-22_lif_54303088_I1.JPG

In recent years, it's become apparent that stem cells will be a key ingredient of future medical treatments. And nowhere is the use of these 'building block cells' more important than through their amazing ability to repair the heart.

The Heart Cells Foundation charity (heartcellsfoundation.com) is funding pioneering research into potentially lifesaving treatment using a patient's own stem cells as a natural repair system to treat heart problems, including heart disease, heart failure and cardiomyopathy. Trials show the stem cells may be able to restore damaged heart tissue, and the treatment is being offered to selected heart failure patients at the Heart Cells Foundation-funded Compassionate Unit at St Bartholomew's Hospital in London. It's the first treatment of its kind in Europe.

Professor Metin Avkiran, associate medical director at the British Heart Foundation (bhf.org.uk), says: "Our hearts have very limited ability to repair themselves following damage from heart attacks and other conditions. This can lead to heart failure, an incurable condition with a worse survival rate than many cancers.

"Stem cells hold immense promise in helping repair damaged hearts, and there have been some encouraging results from a number of studies in patients treated with such cells."

And consultant cardiologist Professor Anthony Mathur, principal investigator on the Heart Cells Foundation's stem cell trials at St Bart's Hospital, adds: "This is a truly exciting field from the perspective of patients healing themselves with their own cells. Clinical trials have helped our understanding of how stem cells may be isolated and then injected into the heart, to promote healing, and we've been fortunate in our own trials to see so many patients with heart disease report improvement in their symptoms, having previously been told no more could be done.

"With more than one million people suffering with heart disease and failure in the UK, the need for treatment in this field has never been greater. As the clinical trial was so successful, we have now been able to launch The Compassionate Treatment Programme at St Bartholomew's Hospital, which is the first of its kind in the UK, and will enable us to treat patients suffering from heart disease on compassionate grounds."

Professor Mathur explains more about stem cells and how they could help mend broken hearts ...

What are stem cells?

"Stem cells have a unique ability to transform into any type of cell within the human body, making them an exciting and revolutionary treatment option for a variety of different conditions."

Are stem cells donated or do patients use their own?

"The therapy used in the trials and the Compassionate Unit utilises the patient's own stem cells to repair the heart. Stem cells are extracted from a patient's bone marrow and injected into the damaged area of their heart."

What heart conditions can stem cells treat?

"The programme based at the Barts Heart Centre has focused on the use of autologous stem cell therapy (patient's own cells) and we have conducted four clinical trials over the last 15 years, to understand what role stem cell therapy has in treating heart disease. Our results have identified individuals with dilated cardiomyopathy and ischaemic heart disease achieve the most benefit from stem cell therapy."

How do stem cells help?

"In the specific case of heart failure, stem cells initiate a repair process within the heart which improves heart function and also patient symptoms, resulting in an improved quality of life. It's the same process of cardiac repair for heart disease and cardiomyopathy. They are still likely to be part of a reparative process. This treatment is no longer just in the laboratory, it's improving the lives of patients all over the UK."

What research has been completed?

"In the programme's last clinical trial, patients with heart disease were treated using stem cell therapy and analysed against a placebo group. Results proved the patients' hearts had started to pump more efficiently, and patients also reported an overall improved physiological and psychological state, enabling them to return to a lifestyle nearer to normal."

What's the next step for heart stem cell research?

"We are now raising funds (approximately 8m) to conduct a larger Phase III trial to consolidate these findings. If successful, the final trial will provide further evidence to the NHS to consider adopting this therapy as standard care. Currently, until the Phase III trials are complete, we have the compassionate programme only. The future timing depends on funding the Phase III trials, which have been planned and are ready to go."

Can someone with a heart problem get stem cell treatment at the moment?

"Stem cell therapy isn't currently available within the NHS. However, the Compassionate Unit at St Bartholomew's allows us to treat patients from across the UK suffering with heart failure due to ischaemic heart disease of dilated cardiomyopathy, who have no further treatment options available to them. The Compassionate Unit is happy to receive enquiries from patients, GPs, cardiologists and healthcare professionals."

Belfast Telegraph

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How stem cells are helping to mend broken hearts - Belfast Telegraph

Iran sends US list of names for its proposed prisoner swap – Stars and Stripes

By Stell Cell Research

TEHRAN, Iran Iran's foreign ministry said Monday it has sent the United States a list of names it is demanding in a proposed prisoner swap, opening a potential new channel with Washington amid recent growing tensions.

Iran did not detail the names it relayed, but Foreign Minister Mohammad Javad Zarif said he hoped to hear soon "good news" about the release of Iranian scientist Masoud Soleimani. U.S. federal authorities arrested Soleimani last year on charges that he had violated trade sanctions by trying to have biological material brought to Iran. Zarif said he raised the issue last month in his visit to New York to attend the U.N. General Assembly, according to the semi-official Fars news agency.

Ministry spokesman Abbas Mousavi said the Islamic Republic has relayed which Iranians should be included in the suggested swap with the United States and other Western nations. Iran holds several American nationals and did not detail whom it would consider freeing.

"We have handed over a list of names (to the United States) who must be freed," Mousavi said, in a briefing with reporters. "We hope that these efforts, if paired with good will, would pay off soon and we would see freedom of Dr. Soleimani and other Iranians from the Americans' captivity."

Iran contends Soleimani and others were detained over what they called "baseless" accusations of bypassing unilateral American sanctions on Iran. It's not clear how many other Iranians the U.S has detained, and there was no immediate American reaction.

Tensions between Iran and the United States have steadily escalated since President Donald Trump pulled the United States out of the 2015 nuclear deal last May and re-imposed sanctions on Iran. Prosecutors in Atlanta got an indictment the following month against Soleimani, who works in stem cell research, hematology and regenerative medicine. U.S. officials revoked his visa and arrested him in October when he landed in Chicago.

The U.S. blames Iran for a series of mysterious oil tanker attacks this year and alleges it carried out last month's attack on the world's largest oil processor in Saudi Arabia, which caused oil prices to spike by the biggest percentage since the 1991 Gulf War.

Iran denies the accusations and has warned any retaliatory attack targeting it will result in an "all-out war," as it has begun enriching uranium beyond the terms of its 2015 nuclear deal. Iran also shot down a U.S. military surveillance drone and seized oil tankers.

A prisoner swap could offer a breakthrough following a pair of conciliatory moves.

A month ago, the U.S. deported Iranian Negar Ghodskani who was brought to the U.S. to face criminal conspiracy charges. She was sentenced to time served for conspiracy to illegally export restricted technology from the U.S. to Iran.

In June, Iran released Nizar Zakka, a U.S. permanent resident from Lebanon who advocated for internet freedom and has done work for the U.S. government. He was sentenced to 10 years on espionage-related charges and was freed after serving less than four years.

However, in May, Iran sentenced former U.S. Navy cook Michael R. White from Imperial Beach, California, to 10 years in prison in Iran, becoming the first American known to be imprisoned there since Trump took office.

Three other American citizens are known to be held in Iran, though Iran does not recognize their dual nationality

Iranian-American Siamak Namazi and his octogenarian father Baquer, a former representative for the U.N. children's agency UNICEF who served as governor of Iran's oil-rich Khuzestan province under the U.S.-backed shah, are both serving 10-year sentences on espionage charges.

Iranian-American art dealer Karan Vafadari and his Iranian wife, Afarin Neyssari, received 27-year and 16-year prison sentences, respectively.

Chinese-American graduate student Xiyue Wang was sentenced to 10 years in prison for allegedly "infiltrating" the country while doing doctoral research on Iran's Qajar dynasty.

Iranian-American Robin Shahini was released on bail in 2017 after staging a hunger strike while serving an 18-year prison sentence for "collaboration with a hostile government." Shahini has since returned to America and is now suing Iran in U.S. federal court.

Former FBI agent Robert Levinson, who vanished in Iran in 2007 while on an unauthorized CIA mission, remains missing. Iran says that Levinson is not in the country and that it has no further information about him, though his family holds Tehran responsible for his disappearance.

Others held with Western ties include Nazanin Zaghari-Ratcliffe, a British-Iranian woman who is serving a five-year prison sentence for allegedly planning the "soft toppling" of Iran's government while traveling with her young daughter. Her daughter left Iran for Britain last week.

____

Heller reported from Dubai, United Arab Emirates.

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Iran sends US list of names for its proposed prisoner swap - Stars and Stripes

Multiple Myeloma Experts, Patients, Advocates and Caregivers Team Up to Hike Through Patagonia – BioSpace

By Stell Cell Research

Since MM4MM began with its first climb in 2016, the program has raised over $2.7 million. All the funds raised go directly to the MMRF to accelerate new treatment options for patients with multiple myeloma.

As a patient founded organization, the MMRF stands together with those who are battling multiple myeloma patients, families, physicians, researchers, and our pharmaceutical partners. This team represents a microcosm of that myeloma community and demonstrates that together, we can collaborate with ever increasing momentum towards a cure, said Paul Giusti, CEO of the Multiple Myeloma Research Foundation. We are thrilled to enter the fifth year of this inspiring program and to have Celgene join us in this effort to raise awareness and critical funds to continue our mission.

The MM4MM team will include four patients living with multiple myeloma:

We are so honored to be a part of yet another hike with the MMRF and Celgene, said Mike Hennessy Jr., president and CEO of MJH Life Sciences, parent company of CURE magazine. This initiative organized by Moving Mountains for Multiple Myeloma not only raises awareness and research funding for multiple myeloma but has brought together the myeloma community to take action and fight for a cure for myeloma patients.

The team will embark on a five-day trek of a lifetime through Patagonia and take on the rewarding and beautiful landscape that includes glaciers, deep valleys and challenging peaks. During this trek, the team will travel through El Chaltn and acclimatize while they experience the mighty range of peaks dominated by Monte Fitz Roy, an 11,020-foot tower with a sheer face of more than 6,000 feet. Next, the team will reach Lago San Martin, where they will traverse the terrain in daily treks, exploring a 10-mile peninsula, climbing to a condor rookery and reaching remote Andean lakes.

Celgene, Cure and the MMRF share an unwavering commitment to improving the lives of patients with multiple myeloma and we are very proud to continue our role in the Moving Mountains for Multiple Myeloma initiative, said Chad Saward, senior director, patient advocacy at Celgene Corp. We are amazed and inspired by all who are participating in this unique awareness program.

To learn more about MM4MM and to donate to multiple myeloma research, click here.

About Moving Mountains for Multiple Myeloma

Moving Mountains for Multiple Myeloma (MM4MM) is a collaboration between CURE Media Group and the Multiple Myeloma Research Foundation (MMRF) to raise awareness and funds for myeloma research. This year, Celgene Corporation and GSK join the effort as sponsors. In addition to Patagonia, the program also led hikes up Mt. Washington and through Iceland in 2019. To date, MM4MM has raised over $2.7 million for myeloma research and included 51 patients with multiple myeloma on 7 climbs. Funds raised go directly to research, supporting the MMRF mission. For more information, visit https://www.themmrf.org/events/.

About Multiple Myeloma

Multiple myeloma (MM) is a cancer of the plasma cell. It is the second most common blood cancer. An estimated 32,110 adults (18,130 men and 13,980 women) in the United States will be diagnosed with MM in 2019 and an estimated 12,960 people are predicted to die from the disease. The five-year survival rate for MM is approximately 50.7%, versus 31% in 1999.

About the Multiple Myeloma Research Foundation

A pioneer in precision medicine, the Multiple Myeloma Research Foundation (MMRF) seeks to find a cure for all multiple myeloma patients by relentlessly pursuing innovations that accelerate the development of precision treatments for cancer. Founded in 1998 by Kathy Giusti, a multiple myeloma patient, and her twin sister Karen Andrews as a 501(c)(3) nonprofit organization, the MMRF has created the business model around cancerfrom data to analytics to the clinic. The MMRF identifies barriers and then finds the solutions to overcome them, bringing in the best partners and aligning incentives in the industry to drive better outcomes for patients. Since its inception, the organization has collected thousands of samples and tissues, opened nearly 100 trials, helped bring 10 FDA-approved therapies to market, and built CoMMpass, the single largest genomic dataset for any cancer. Today, the MMRF is building on its legacy in genomics and is expanding into immune-oncology, as the combination of these two fields will be critical to making precision medicine possible for all patients. The MMRF has raised nearly $500 million and directs nearly 90% of the total funds to research and related programs. To learn more, visit http://www.themmrf.org.

About CURE Media Group

CURE Media Group is the leading resource for cancer updates, research and education. It combines a full suite of media products, including its industry-leading website, CUREtoday.com; innovative video programs, such as CURE Connections; a series of widely attended live events; and CURE magazine, which reaches over 1 million readers, as well as the dynamic website for oncology nurses, OncNursingNews.com, and its companion publication, Oncology Nursing News. CURE Media Group is a brand of MJH Life Sciences, the largest privately held, independent, full-service medical media company in the U.S. dedicated to delivering trusted health care news across multiple channels.

View source version on businesswire.com: https://www.businesswire.com/news/home/20191022006008/en/

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Multiple Myeloma Experts, Patients, Advocates and Caregivers Team Up to Hike Through Patagonia - BioSpace

Global consortium formed to combat unproven cell banking services – Drug Target Review

By Induced Pluripotent Stem Cells

The International Society for Cell and Gene Therapy has created a group to tackle the rising number of commercial cell bank services that are misleading patients.

The International Society for Cell and Gene Therapy (ISCT) has announced the formation of a global consortium to combat the growing number of unproven commercial cell banking services.

The group will be made up of leading professional and education societies, including, among others:

The partnership has been formed following the ISCTs publication of its patient advice and concern on unproven T cell preservation services. These facilities comprise the banking of T cells, dental cells and cells for the derivation of induced pluripotent stem cells for potential therapeutic uses.

these cell banking services can deceive patients using tokens of scientific legitimacy

A joint statement from the ISCT and the consortium partners commented on certain commercial cell banking services and their lack of support from current scientific evidence. Furthermore, the society says that these cell banking services are unable to declare that cells they preserve may ever be appropriate for clinical usage or for manufacturing purposes.

The ISCT emphasises that there is no clear pathway to legitimate clinical use. As such, any parties offering these services commercially to patients is premature, misleading and drives false hope.

Any patients using these services are therefore prevented from giving full and valid informed consent, according to the ISCT.

The society highlights that these cell banking services can deceive patients using tokens of scientific legitimacy that suggest a stronger scientific basis than currently exists. These include endorsements from individuals or scientific advisory boards that may not fully support the specific products, links to scientific articles and references to ongoing clinical trials.

ISCTs raison detre is to lead the industry in supporting scientifically validated cell and gene therapies. As a result, ISCT will continue to welcome all innovations, including cell banking approaches, that increase the number of patients who can benefit from these therapies, said Bruce Levine,President-Elect, ISCT.However, ISCT also leads industry action on unproven cell therapies and services in the cell and gene sector.

This is why ISCT has forged a consortium throughout the industry against the marketing of speculative cell banking services that do not have appropriate pre-clinical and clinical evidence and a plausible pathway to the clinical use of banked cells. We collectively believe these banks have the potential to be detrimental to the future development of cell and gene therapies.

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Global consortium formed to combat unproven cell banking services - Drug Target Review

Bloomberg Philanthropies, Johns Hopkins University School of Medicine, and The New York Stem Cell Foundation Research Institute Announce an…

By Induced Pluripotent Stem Cells

NEW YORK, Oct. 22, 2019 /PRNewswire/ -- Bloomberg Philanthropies, Johns Hopkins University School of Medicine (JHUSOM), and The New York Stem Cell Foundation (NYSCF) Research Institute today announced an initiative to fundamentally advance and expand the science of precision medicine, in which diagnostic disease markers are defined with pinpoint accuracy to help researchers understand disease pathways and customize therapeutic approaches. The collaboration will combine the renowned clinical and medical expertise of Johns Hopkins with the unique stem cell technologies and research capabilities of the NYSCF Research Institute to accelerate Hopkins' pioneering Precision Medicine Initiatives.

"Johns Hopkins is working intensively to realize the great promise of precision medicine for all those in our care, locally and globally," said Johns Hopkins President Ronald J. Daniels. "This significant new collaboration with Bloomberg Philanthropies and NYSCF moves us ever closer to that aim as we join together our far-reaching research capacities to advance knowledge and deliver better health outcomes for populations and people around the world."

This collaboration will also establish an unprecedented cache of human disease models available to researchers worldwide thus promoting the real world application of precision medicine and driving a new paradigm for understanding and improving the approach to human disease.

"Bloomberg Philanthropies' mission is to ensure better, longer lives for the greatest number of people," said Michael R. Bloomberg, founder of Bloomberg LP and Bloomberg Philanthropies. "For years, Johns Hopkins University and the New York Stem Cell Foundation have shared that mission and we're honored to deepen our partnerships with them as they explore new, innovative ways to save lives through the application of precision medicine."

Diseases manifest themselves differently in different patients. To understand the basis of these differences and to tailor treatments for specific patients, researchers need more accurate biological tools. Stem cell models provide a "biological avatar" of the patient from which they were created, allowing scientists and clinicians to better understand, define, and account for differences in individual patients and groups of patients.

The new initiative will use induced pluripotent stem cells to study disease characteristics in subgroups of patients, identifying markers that lead to varying disease manifestations. For example, by examining stem cells from seemingly similar patients with different forms of multiple sclerosis, we may be able to better understand the full range of disease mechanisms and pathways.

The Johns Hopkins Precision Medicine Initiative already includes 16 Precision Medicine Centers of Excellence (PMCOE), each focusing on a specific disease, and is now working to develop 50 Precision Medicine Centers in the next five years. Johns Hopkins believes that this advancement in the study and application of precision medicine has the potential to transform the diagnosis and management of many diseases.Often, what is now categorized as a single disease is actually made up ofmultiple diseases that display similar symptoms, but require quite different therapies. Using a wide range of data sources, precision medicine seeks to better elucidate these differences, so that doctors can treat patients with precisely targeted therapies. At Johns Hopkins, dozens of researchers are bringing this idea to reality across a spectrum of debilitating and life-altering diseases.

In this collaboration, the process will begin with the full consent of patients in JHUSOM PMCOEs who wish to participate. Biological samples from the JHUSOM PMCOEs will be collected by the NYSCF Research Institute where scientists will create stem cell models of disease using the NYSCF Global Stem Cell Array, the world's first end-to-end automated system for generating human stem cells in a parallel, highly controlled process.Integrating robotics and machine learning, NYSCF's technology reprograms skin or blood cells into stem cells, differentiates them into disease-relevant cell types, and performs genome editing to unravel the genetic basis of disease.

"The NYSCF Research Institute has invented and scaled the most advanced methods of human cell manipulation, which is critical for studying disease at the level of the individual patient," explained NYSCF CEO Susan L. Solomon. "By combining our capabilities with Johns Hopkins' extensive clinical data and expertise, we will be able to develop effective, personalized therapies for patients suffering from diseases with a high unmet need."

The stem cells generated by NYSCF will be used to research and drive effective therapeutic and diagnostic development in a wide range of diseases that include, but are not limited to, Multiple Sclerosis, Alzheimer's, chronic renal failure, and cancers of the lung, breast, prostate, pancreas, and bladder. These stem cell lines will reside in the NYSCF Repository and serve as an extraordinary resource in perpetuity for the disease research community. This vast collection will allow scientists unprecedented insights into the biochemical and genetic mechanisms underlying different diseases and subtypes thereof, thereby illuminating avenues for effective, tailored interventions.

"Stem cell science holds enormous potential for the treatment of a wide range of diseases," said Paul B. Rothman, dean of the School of Medicine and CEO of Johns Hopkins Medicine. "By combining this approach with Johns Hopkins' groundbreaking work on precision medicine, we are creating a scientific powerhouse that will help us advance medicine and science at an even faster pace. I am excited to see the discoveries and innovations that will be produced by this collaboration."

About Bloomberg PhilanthropiesBloomberg Philanthropies invests in 510 cities and 129 countries around the world to ensure better, longer lives for the greatest number of people. The organization focuses on five key areas for creating lasting change: Arts, Education, Environment, Government Innovation, and Public Health. Bloomberg Philanthropies encompasses all of Michael R. Bloomberg's giving, including his foundation and personal philanthropy as well as Bloomberg Associates, a pro bono consultancy that works in cities around the world. In 2018, Bloomberg Philanthropies distributed $767 million. For more information, please visitbloomberg.orgor follow us on Facebook, Instagram, YouTube, and Twitter.

About The New York Stem Cell Foundation Research Institute The New York Stem Cell Foundation (NYSCF) Research Institute is an independent non-profit organization accelerating cures and better treatments for patients through stem cell research. The NYSCF global community includes over 180 researchers at leading institutions worldwide, including the NYSCF Druckenmiller Fellows, the NYSCF Robertson Investigators, the NYSCF Robertson Stem Cell Prize Recipients, and NYSCF Research Institute scientists and engineers. The NYSCF Research Institute is an acknowledged world leader in stem cell research and in developing pioneering stem cell technologies, including the NYSCF Global Stem Cell Array and in manufacturing stem cells for scientists around the globe. NYSCF focuses on translational research in an accelerator model designed to overcome barriers that slow discovery and replace silos with collaboration. For more information, visit http://www.nyscf.org or follow us on Twitter, Facebook, and Instagram.

Press Contacts:

The New York Stem Cell Foundation Research Institute David McKeon dmckeon@nyscf.org 212-365-7440

Johns Hopkins University School of Medicine Vanessa Wasta wasta@jhmi.edu

SOURCE The New York Stem Cell Foundation

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Bloomberg Philanthropies, Johns Hopkins University School of Medicine, and The New York Stem Cell Foundation Research Institute Announce an...

Dr. Peggy Goodell elected to National Academy of Medicine – Baylor College of Medicine News

By Uncategorized

Dr. Margaret Peggy Goodell, chair of the Department of Molecular and Cellular Biology at Baylor College of Medicine, has been elected to the National Academy of Medicine. She is among 100 new members, including 10 international members, announced today at the Academys annual meeting in Washington, D.C.

Dr. Goodells long career in regenerative medicine proves she is well-deserving of this distinction, said Dr. Paul Klotman, president, CEO and executive dean of Baylor. She is a renowned scientist who has made important contributions to the field of stem cell biology, particularly genetic and epigenetic regulation of self-renewal and differentiation of hematopoietic stem cells.

Goodell is known for discovering a novel method to isolate adult stem cells. At Baylor, she serves as a professor in several departments, including pediatrics hematology-oncology and molecular and human genetics and in the Center for Cell and Gene Therapy, as well as programs in integrative molecular and biomedical sciences, development biology and translational biology and molecular medicine. She is the director of the Stem Cells and Regenerative Medicine Center at Baylor and holds the Vivian L. Smith Chair in Regenerative Medicine. She is co-leader of the Cancer Cell and Gene Therapy Program in the Dan L Duncan Comprehensive Cancer Center at Baylor.

I am extremely honored by election to the National Academy of Medicine. This distinction represents recognition of my work, as well as the contributions of the many members of my laboratory who have worked with me over the past 20 years, Goodell said. I am looking forward to the opportunity to contribute to the NAM on relevant national science and medicine issues.

Election to the National Academy of Medicine is considered one of the highest honors in the fields of health and medicine and recognizes individuals who have demonstrated outstanding professional achievement and commitment to service. New members are elected by current members through a process that recognizes individuals who have made major contributions to the advancement of the medical sciences, health care and public health.

Goodell joins the following group of 13 distinguished scientists from Baylor College of Medicine in the National Academy of Medicine:

Dr. Arthur L. BeaudetDr. Dennis M. BierDr. Malcolm K. BrennerDr. William R. BrinkleyDr. C. Thomas CaskeyDr. Mary K. EstesDr. Richard A. GibbsDr. Peter J. HotezDr. Brendan LeeDr. James R. LupskiDr. Bert W. OMalleyDr. Cheryl Lyn WalkerDr. Huda Y. Zoghbi

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Dr. Peggy Goodell elected to National Academy of Medicine - Baylor College of Medicine News

New Organelle That Helps Prevent Cancer Discovered Inside Our Cells – University of Virginia

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Scientists at theUniversity of Virginia School of Medicinehave discovered a strange neworganelle inside our cells that helps to prevent cancer by ensuring that genetic material is sorted correctly as cells divide.

The researchers have connected problems with the organelle (a subcellular structure) to a subset ofbreast cancer tumorsthat make lots of mistakes when segregating chromosomes. Excitingly, they found their analysis offered a new way for doctors to sort patient tumors as they choose therapies. Theyhope these insights will allow doctors to better personalize treatments to best benefit patients sparing up to 40% of breast cancer patients, for example, a taxing treatment that wont be effective.

Some percentage of women get chemotherapy drugs for breast cancer that are not very effective. They are poisoned, in pain and their hair falls out, so if it isnt curing their disease, then thats tragic, said researcher P. Todd Stukenberg of UVAs Department of Biochemistry and Molecular Genetics and theUVA Cancer Center. One of our goals is to develop new tests to determine whether a patient will respond to a chemotherapeutic treatment, so they can find an effective treatment right away.

The organelle Stukenberg and his team have discovered is essential, but ephemeral. It forms only when needed to ensure chromosomes are sorted correctly and disappears when its work is done. Thats one reason scientists havent discovered it before now.

Another reason is its mind-bending nature: Stukenberg likens it to a droplet of liquid that condenses within other liquid. That was the big wow moment, when I saw that on the microscope, he said.

These droplets act as mixing bowls, concentrating certain cellular ingredients to allow biochemical reactions to occur in a specific location. Whats exciting is that cells have this new organelle and certain things will be recruited into it and other things will be excluded, Stukenberg said. The cells enrich things inside the droplet and, all of a sudden, new biochemical reactions appear only in that location. Its amazing.

Its tempting to think of the droplet like oil in water, but its really the opposite of that. Oil is hydrophobic it repels water. This new organelle, however, is more sophisticated.

Its more of a gel, where cellular components can still go in and out, but it contains binding sites that concentrate a small set of the cells contents, Stukenberg explained. Our data suggests this concentration of proteins is really important. I can get complex biochemical reactions to occur inside a droplet that Ive been failing to reconstitute in a test tube for years. This is the secret sauce Ive been missing.

While its been known for about eight years that cells make such droplets for other processes, it was unknown that they make them on chromosomes during cell division. Stukenberg believes these droplets are very common and more important than previously realized.

I think this is a general paradigm, he said. Cells are using these non-membranous organelles to regulate much of their work.

In addition to helping us understand mitosis how cells divide Stukenbergs new discovery also sheds light on cancer and how it occurs. The organelles main function is to fix mistakes in tiny microtubules that pull apart chromosomes when cells are dividing. That ensures each cell winds up with the correct genetic material. In cancer, though, this repair process is defective, which can drive cancer cells to become more aggressive.

Stukenberg has also developed tests to measure the amount of chromosome mis-segregation in tumors, and he hopes that this might allow doctors to pick the proper treatment to give cancer patients. We have a way to identify the tumors where the cells are mis-segregating chromosomes at a higher rate, he said. My hope is to identify the patients where treatments such as [chemotherapy medication] paclitaxel are going to be the most effective.

Having looked at breast cancer already, he next plans to examine the strange organelles role in colorectal cancer.

Stukenberg and his colleagues described their latest findings in the scientific journal Nature Cell Biology. The research team consisted of Prasad Trivedi, Francesco Palomba, Ewa Niedzialkowska, Michelle A. Digman, Enrico Gratton and Stukenberg.

The research was supported by the National Institutes of Health, grants R01GM124042, R24OD023697 and P41-GM103540; and the National Science Foundation, grant MCB-1615701.

To keep up with the latest medical research news from UVA, subscribe to theMaking of Medicineblog.

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New Organelle That Helps Prevent Cancer Discovered Inside Our Cells - University of Virginia

Capricor Therapeutics to Host Key Opinion Leader Call on the Role of CAP-1002 for the Treatment of Duchenne Muscular Dystrophy (DMD) – BioSpace

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LOS ANGELES, Oct. 21, 2019 (GLOBE NEWSWIRE) -- Capricor Therapeutics (Nasdaq: CAPR), a clinical-stage biotechnology company focused on the development of first-in-class biological therapeutics for the treatment of Duchenne muscular dystrophy (DMD) and other rare disorders, announced today that it will host a Key Opinion Leader (KOL) call on the role of CAP-1002 in the of treatment of Duchenne Muscular Dystrophy (DMD) on Thursday, October 24th at 10:30am Eastern Time.

The call will feature a presentation by KOL Craig M. McDonald, MD, UC Davis Health, and principal investigator of Capricors HOPE-2 trial who will provide an overview of Duchenne Muscular Dystrophy (DMD), its progression, current treatment options, and new treatments in development for the disease. Dr. McDonald will be available to answer questions at the conclusion of the call.

Capricors management team will also provide an overview of the Companys lead candidate, CAP-1002, a cell therapy that is currently in clinical development for the treatment of Duchenne muscular dystrophy (DMD). CAP-1002 consists of allogeneic cardiosphere-derived cells, or CDCs, a unique population of cells that contains cardiac progenitor cells. CAP-1002 has been shown to exert potent immunomodulatory activity and alter the immune systems activity to encourage cellular regeneration. Data from the pre-specified interim analysis demonstrated that teens and young men in the advanced stages of DMD saw improvements in skeletal, pulmonary, and cardiac measurements after receiving multiple doses of CAP-1002. Specifically, patients showed improvements in the Performance of the Upper Limb (PUL), a tool specifically designed for assessing high (shoulder), mid (elbow) and distal (wrist & hand) function, with a conceptual framework reflecting the progression of weakness in ambulant and non-ambulant patients.

The FDA has granted Capricors CAP-1002 (Regenerative Medicine Advanced Therapy Designation) RMAT and Orphan Drug Designation, for which the FDA has also granted a Rare Pediatric Disease Designation.

Craig M. McDonald, MD is currently a Professor of Medicine and the Chair of the Department of Physical Medicine & Rehabilitation at UC Davis Health. He received his M.D. and M.R.M. from the University of Washington School of Medicine as well as his A.B. in Human Biology from Stanford University. Dr. McDonald is also board-certified in neuromuscular medicine and pediatric rehabilitation medicine. He is an internationally recognized expert in clinical management, rehabilitation, and precision therapeutics for children and adults with neuromuscular diseases. Dr. McDonald has been a pioneer in the development of novel outcome measures for clinical trials focused on disabled populations. He is widely known for his expertise in the treatment and evaluation of children and young adults with Duchenne muscular dystrophy and other neuromuscular diseases. Dr. McDonald serves as director and principal investigator of UC Davis' successfully renewed NINDS-funded site in the NeuroNEXT Neurosciences Clinical Trials National Consortium (one of two NeuroNEXT sites on the West Coast). Dr. McDonald is also the director of rehabilitation services at Shriners Hospital for Children - Northern California.

About HOPE-2HOPE-2 is a randomized, double-blind, placebo-controlled, Phase II clinical trial of the companys lead investigational therapy, CAP-1002 in steroid-treated boys and young men who are in advanced stages of DMD. The study protocol called for treatment via intravenous delivery with either CAP-1002 (150 million cells per infusion) or placebo every 3 months.

About Duchenne Muscular DystrophyDuchenne muscular dystrophy is a devastating genetic disorder that causes muscle degeneration and leads to death, generally before the age of 30, most commonly from heart failure. It occurs in one in every 3,600 live male births across all races, cultures and countries. Duchenne muscular dystrophy afflicts approximately 200,000 boys and young men around the world. Treatment options are limited, and there is no cure.

About CAP-1002CAP-1002 consists of allogeneic cardiosphere-derived cells, or CDCs, a proprietary population of cells that contains cardiac progenitor cells that has been shown in pre-clinical and clinical studies to exert potent immunomodulatory activity, and is being investigated for its potential to modify the immune systems activity to encourage cellular regeneration. CDCs have been the subject of over 100 peer-reviewed scientific publications and have been administered to approximately 150 human subjects across several clinical trials.

About Capricor TherapeuticsCapricor Therapeutics, Inc. (CAPR) is a clinical-stage biotechnology company focused on the discovery, development and commercialization of first-in-class biological therapeutics for the treatment of rare disorders. Capricors lead candidate, CAP-1002, is an allogeneic cell therapy that is currently in clinical development for the treatment of Duchenne muscular dystrophy. Capricor is also exploring the potential of CAP-2003, a cell-free, exosome-based candidate, to treat a variety of disorders. HOPE-Duchenne, Capricors Phase I/II trial was funded in part by the California Institute for Regenerative Medicine. For more information, visit http://www.capricor.com.Keep up with Capricor on social media: http://www.facebook.com/capricortherapeutics, http://www.instagram.com/capricortherapeutics/ and https://twitter.com/capricor

Cautionary Note Regarding Forward-Looking StatementsStatements in this press release regarding the efficacy, safety, and intended utilization of Capricor's product candidates; the initiation, conduct, size, timing and results of discovery efforts and clinical trials; the pace of enrollment of clinical trials; plans regarding regulatory filings, future research and clinical trials; regulatory developments involving products, including the ability to obtain regulatory approvals or otherwise bring products to market; plans regarding current and future collaborative activities and the ownership of commercial rights; scope, duration, validity and enforceability of intellectual property rights; future royalty streams, revenue projections; expectations with respect to the expected use of proceeds from the recently completed offerings and the anticipated effects of the offerings, and any other statements about Capricor's management team's future expectations, beliefs, goals, plans or prospects constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including statements containing the words "believes," "plans," "could," "anticipates," "expects," "estimates," "should," "target," "will," "would" and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward-looking statements. More information about these and other risks that may impact Capricor's business is set forth in Capricor's Annual Report on Form 10-K for the year ended December 31, 2018 as filed with the Securities and Exchange Commission on March 29, 2019, and as amended by its Amendment No. 1 to Annual Report on Form 10-K/A filed with the Securities and Exchange Commission on April 1, 2019, in its Quarterly Report on Form 10-Q for the quarterly period ended June 30, 2019, as filed with the Securities and Exchange Commission on August 8, 2019, and in its Registration Statement on Form S-3 as filed with the Securities and Exchange Commission on October 24, 2018, and as amended by its Amendment No. 1 to Form S-3 filed with the Securities and Exchange Commission on July 17, 2019, together with prospectus supplements thereto. All forward-looking statements in this press release are based on information available to Capricor as of the date hereof, and Capricor assumes no obligation to update these forward-looking statements.

CAP-1002 is an Investigational New Drug and is not approved for any indications. CAP-2003 has not yet been approved for clinical investigation.

For more information, please contact:

AJ Bergmann, Chief Financial Officer+1-310-358-3200abergmann@capricor.com

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Capricor Therapeutics to Host Key Opinion Leader Call on the Role of CAP-1002 for the Treatment of Duchenne Muscular Dystrophy (DMD) - BioSpace

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