Stem cell initiative: Save lives and energize the economy – Capitol Weekly

Opinion

by BOB KLEIN posted 07.09.2020

In our new financial reality, our state and you as voters are faced with tough decisions. Come November, you will decide the fate of Californias stem cell institute. This decision has never been more important to the future of Californias health care, for the patients and their families, than it is now.

Californians overwhelmingly approved the states first stem cell initiative in 2004, with nearly 60% of the vote, and widespread support from patient advocacy organizations, labor, business and elected officials. The 2004 initiative established the California Institute for Regenerative Medicine (CIRM) to fund medical research and the development of new treatments and cures.

If Californians do not pass Proposition 14, vital lifesaving research will come to a halt.

CIRM funding has advanced research and therapy development for more than 75 different diseases and conditions, more than 90 clinical trials, more than 1,000 medical projects at 70 institutions across California and nearly 3,000 published medical discoveries. This investment has already saved and improved lives, including a high school student who was paralyzed in a diving accident and was able to regain function in his upper body and go on to college, a mother who went blind from a genetic disease has had some of her eyesight restored, two FDA-approved cancer treatments are already saving lives, and many more.

As funding for Californias stem cell research program has now run out, Californians have a critical opportunity to pass the 2020 stem cell initiative Proposition 14 to continue to advance lifesaving research and treatment development. Proposition 14 just recently qualified for the November ballot with early support from more than 65 patient advocacy organizations, the University of California and Nobel Prize winners.

If Californians do not pass Proposition 14, vital lifesaving research will come to a halt. Medical discoveries wont be able to progress to clinical trials, delaying lifesaving and life-changing treatments for cancer, diabetes, heart disease, Alzheimers, Parkinsons, infectious diseases like COVID-19 and more for years or decades.

Additionally, not passing Proposition 14 would eliminate a critical economic and jobs stimulus for our state.

Arguably, our state leaders are rightfully concerned about the state budget amid the current landscape. But as we enter a period of economic crisis, this bond measure will act as an economic recovery and jobs stimulus.

It is not a tax; it is a general obligation bond that will be repaid by the state, over decades, beginning in 2026.

Proposition 14 will increase the amount of state funds available to tackle other issues for the next 10 years by increasing state tax revenues through 2030 potentially providing more state revenue than the cost of bond payments during that time. To date, Californias stem cell research program has generated $10.7 billion in increased state economic activity and hundreds of millions in additional state revenues. Californias stem cell research program also creates tens of thousands of jobs at every level from lab technicians and maintenance workers to nurses and physicians.

Proposition 14 is exactly the kind of long-term investment we should make now to rebuild our economy. The initiative was specifically designed to be fiscally responsible. It is not a tax; it is a general obligation bond that will be repaid by the state, over decades, beginning in 2026 a full five years after its passage.

We cant afford not to fund Proposition 14.

Chronic diseases are the leading cause of death and the leading driver of annual health care spending and bankruptcies. In California alone, more than 30% of the states budget is spent on health care. With this cost rising every year, it is a growing strain on California families and our state budget.

If we hesitate to fund proper research to develop cures for chronic illnesses, our health care costs will financially drain California families, as well as our struggling state budget. Reducing the cost of treating just 6 of 80 major chronic diseases or injuries by 1-2% would pay for the Initiative twice over.

The decision you make in November will have lasting impacts on the state funds available to tackle priorities California needs to address now, and in the future, including housing, education, or the environment. Proposition 14 will generate additional revenue to help address these issues now, and potentially save California tens of billions for the future.

Proposition 14 will cost the state an average of less than $5 per person, per year about the cost of a bottle of aspirin. That is a small price to pay to potentially save millions of lives and tens of billions of dollars in health care costs in the coming decades. At the end of the day, Proposition 14 could save your life or the life of someone you love how can we afford not to make this investment? Editors Note: Bob Klein is chairman of Californians for Stem Cell Research, Treatments and Cures.

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Stem cell initiative: Save lives and energize the economy - Capitol Weekly

Researchers at U of T use stem cells to grow functional blood vessel cells found in liver – News@UofT

An inter-disciplinary team of researchers, funded by the University of Torontos Medicine by Design,hasgeneratedfunctional blood vessel cells found in the liver from stem cells a discovery thatoffersan opportunityto study the rolethe cellsplayin liver developmentand diseaseprogression, and which couldlead tonew therapies to treat hemophilia A.

Thestudy, titledGeneration of Functional Liver Sinusoidal Endothelial Cells from Human Pluripotent Stem Cell-Derived Venous Angioblasts,waspublished this week in Cell Stem Cell. Itrepresents a collaborative effort betweenbasic and clinical researchersat U of T and the University Health Network (UHN)with expertise in stem celland computationalbiology,human liver physiology and functionand liver transplantation.

It alsodraws onprevious Medicine by Design-funded research that led to the creationin 2018of the first single-cell map of the human liver.

By combining insights from developmental biology and liver anatomy with thecellatlasof the human liver,we were able to generateand validatefunctional human liver vasculature from stem cells, saysBlair Gage,apost-doctoral researcher at the McEwen Stem Cell Institute at UHNand lead author ofthestudy.Nowwe canmove forwardto use these liver endothelial cells tobetterunderstandtheir role in liver functionand to develop new therapies to treat disorderssuch ashemophilia A.

Theinterdisciplinary researchteam also includes:JeffLiu,research associate atU of TsDonnelly Centrefor Cellular andBiomolecularResearch;Brendan Innes, a PhD candidate at the Donnelly Centre and in thedepartment ofmolecular genetics in the Faculty of Medicine;Sonya MacParland, scientist in themulti-organ transplant program at theToronto General Hospital Research Institute andan assistant professor in U of Ts departments of immunology andlaboratory medicine and pathobiology; Ian McGilvray,senior scientist at themulti-organ transplant program at theToronto General Hospital Research Institute and a professorinU of Ts department of surgery;Gary Bader, professor at the Donnelly Centreandthedepartment ofmolecular genetics; andGordon Keller,director andsenior scientist at theMcEwen Stem Cell Institute at UHNandprofessor in U of Ts department of medical biophysics.

Researchersinthe Keller lab had the goal of generatinga functional liver vasculature cell type known as liver sinusoidal cells (LSECs)fromhuman pluripotent stem cells (hPSCs) cells that can self-renew and have the potential to turn into any other cell type in the human body. LSECs are essential for normal liver function and represent the main source of factor VIII,a blood-clotting protein that is missing or defective in patients with hemophilia A.

However, the teamhad todemonstratethatthecellstheyhadmadein the labhad thesamespecializedgenetic and functionalfeaturesasthose in thehuman liver.So they turned to the work of MacParland, Bader and McGilvray,whoin the first phase ofMedicine by Designs team projectfundingdescribed amolecularmap of the cell types in the adult liver.Thatresearchhascontributed totheHuman Cell Atlasan international effort to create comprehensive reference maps of all human cellsand last yearattracted follow-on funding from the Chan Zuckerberg Initiative.

This paper uses our human liver map as a guide to know if the cells beinggeneratedaretherightones through collaboration with Gary Baders group, says MacParland.The work really highlights thestrengthof MedicinebyDesignin bringingtogether researchers from multiple institutionstofocus on a common goal.

With Bader and Lius help, Keller lab researchers were able to use the MacParlandhuman livermapto show that thehPSC-derivedendothelialcellsthey had generatedshared manyof thefeatures found innormal liver vasculature. The Keller lab team then brought Innes on board toformat thedatafromthehPSC-derived LSECsfor the research communitytoeasilyexplorethe molecular profile of these cells.

This research was supported by Medicine by Design, which receives funding from the federal governmentsCanada First Research Excellence Fundand by theCanadian Institutes of Health Research.

The work continues in a current Medicine by Design-funded team projectled by Kellerthat aims to make other key liver cell types and put together the pieces to get functional tissueswith the goal of developing new cell-based therapies for liver-related diseases.That project is part ofanew $20-million round of team project fundingthat Medicine by Design announced late last year.

Medicine by Designbrings togetherinvestigatorsfromdifferent disciplinesatU of T and its affiliated hospitals to advance new discoveries in regenerative medicine and accelerate them toward clinical impact.Medicine by Designwill host ameeting of the Human Cell Atlass Development and Pediatric Atlasin July 2021in Toronto.

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Researchers at U of T use stem cells to grow functional blood vessel cells found in liver - News@UofT

Orchard Therapeutics and MolMed Announce Extension of Gene Therapy Manufacturing Collaboration – BioSpace

BOSTON and LONDON and MILAN, Italy, July 09, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, and MolMed S.p.A (MLMD.MI), one of the companys principal contract development and manufacturing partners, today announced that they have extended their collaboration initiated in April 2018 for a period of five years through June 2025.

With the extension of the collaboration, MolMed will continue to support activities related to the development and manufacturing of vectors and drug products for several of Orchards investigational ex vivo hematopoietic stem cell (HSC) gene therapies in the upcoming years, including OTL-200 for metachromatic leukodystrophy (MLD) and OTL-103 for Wiskott Aldrich syndrome (WAS), as well as for additional pipeline programs including OTL-203 for mucopolysaccharidosis type I (MPS-I). MolMed is the first company to have obtained good manufacturing practice (GMP) authorization for the gene and cell therapy markets in Europe and is the manufacturer for Strimvelis, Orchards ex vivo HSC gene therapy for severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID) and the first such treatment approved by the European Medicines Agency (EMA).

We are looking forward to continuing to build and expand upon our partnership with MolMed, who have supported the progression of many of our programs since their earliest clinical development stages, said Frank Thomas, president and chief operating officer of Orchard. Their expertise in gene therapy manufacturing, coupled with their deep knowledge of our programs, will be invaluable as our therapies for MLD and WAS approach anticipated approval and commercialization in Europe and across the globe.

Luca Alberici, MolMed's chief business officer, added, "We are pleased to have strengthened our collaboration with Orchard to support them in their mission of bringing potentially transformative therapies to those suffering from severe rare diseases. After being Orchards exclusive manufacturer for Strimvelis, we are looking forward to supporting their manufacturing needs for additional programs both in clinical trials and in potential commercial applications following the anticipated approval of OTL-200 for MLD in Europe later this year.

OTL-200 for MLD is currently under review by the EMA with a decision expected later this year.

About Orchard Orchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit http://www.orchard-tx.com, and follow us on Twitter and LinkedIn.

About MolMed MolMed S.p.A. is a biotechnology company focused on research, development, manufacturing and clinical validation of novel cell and gene therapies. MolMed, established in 1996, has been listed since March 2008 on the Italian Stock Exchange managed by Borsa Italiana, and has its registered office in Milan, at the Biotechnology Department of Ospedale San Raffaele and an operating site at Bresso, at the OpenZone campus.

Availability of Other Information About Orchard Investors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (Twitter and LinkedIn), including but not limited to investor presentations and investor fact sheets, U.S. Securities and Exchange Commission filings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

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. Forward-looking statements include express or implied statements relating to, among other things, Orchards business strategy and goals, 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 or marketing approval of its product candidates, and Orchards expectations concerning its partnership with MolMed. 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, these risks and uncertainties include, without limitation: the severity of the impact of the COVID-19 pandemic on Orchards business, including on clinical development and commercial programs; 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 approved, successfully developed or commercialized; the risk of cessation or delay of any of Orchards ongoing or planned clinical trials; the risk that Orchard may not successfully recruit or enroll a sufficient number of patients for its 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 or the receipt of restricted marketing approvals; the risk of delays in Orchards ability to commercialize its product candidates, if approved; and the risk that Orchard may not receive the expected benefits from its collaboration with MolMed or that Orchard or MolMed will not fully perform under the terms of their collaboration agreement. 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 quarterly report on Form 10-Q for the quarter ended March 31, 2020, as filed with the U.S. Securities and Exchange Commission (SEC) on May 7, 2020, 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.

Orchard Contacts

Investors Renee Leck Director, Investor Relations +1 862-242-0764 Renee.Leck@orchard-tx.com

Media Molly Cameron Manager, Corporate Communications +1 978-339-3378 media@orchard-tx.com

MolMed Contacts

Investor Relations & Communications Department MolMed S.p.A. +39 02 21277.205 investor.relations@molmed.com

Tommasina Cazzato Press Office Community Group +39 345 7357751 tommasina.cazzato@communitygroup.it

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Orchard Therapeutics and MolMed Announce Extension of Gene Therapy Manufacturing Collaboration - BioSpace

Fate Therapeutics Announces FDA Clearance of IND Application for First-ever iPSC-derived CAR T-Cell Therapy – GlobeNewswire

July 09, 2020 08:00 ET | Source: Fate Therapeutics, Inc.

FT819 CAR T-cell Product Candidate Derived from Clonal Master iPSC Line with Novel CD19-specific 1XX CAR Integrated into TRAC Locus

Phase 1 Clinical Study will Evaluate FT819 for Patients with Advanced B-cell Leukemias and Lymphomas

SAN DIEGO, July 09, 2020 (GLOBE NEWSWIRE) -- Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, announced today that the U.S. Food and Drug Administration (FDA) has cleared the Companys Investigational New Drug (IND) application for FT819, an off-the-shelf allogeneic chimeric antigen receptor (CAR) T-cell therapy targeting CD19+ malignancies. FT819 is the first-ever CAR T-cell therapy derived from a clonal master induced pluripotent stem cell (iPSC) line, and is engineered with several first-of-kind features designed to improve the safety and efficacy of CAR T-cell therapy. The Company plans to initiateclinical investigation of FT819for the treatment of patients with relapsed / refractory B-cell malignancies, including chronic lymphocytic leukemia (CLL), acute lymphoblastic leukemia (ALL), and non-Hodgkin lymphoma (NHL).

The clearance of our IND application for FT819 is a ground-breaking milestone in the field of cell-based cancer immunotherapy. Our unique ability to produce CAR T cells from a clonal master engineered iPSC line creates a pathway for more patients to gain timely access to therapies with curative potential, said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. Four years ago, we first set out under our partnership with Memorial Sloan Kettering led by Dr. Michel Sadelain to improve on the revolutionary success of patient-derived CAR T-cell therapy and bring an off-the-shelf paradigm to patients, and we are very excited to advance FT819 into clinical development.

FT819 was designed to specifically address several limitations associated with the current generation of patient- and donor-derived CAR T-cell therapies. Under a collaboration with Memorial Sloan Kettering Cancer Center (MSK) led by Michel Sadelain, M.D., Ph.D., Director, Center for Cell Engineering, and Head, Gene Expression and Gene Transfer Laboratory at MSK, the Company incorporated several first-of-kind features into FT819 including:

The multi-center Phase 1 clinical trial of FT819 is designed to determine the maximum tolerated dose of FT819 and assess its safety and clinical activity in up to 297 adult patients across three types of B-cell malignancies (CLL, ALL, and NHL). Each indication will enroll independently and evaluate three dose-escalating treatment regimens: Regimen A as a single dose of FT819; Regimen B as a single dose of FT819 with IL-2 cytokine support; and Regimen C as three fractionated doses of FT819. For each indication and regimen, dose-expansion cohorts of up to 15 patients may be enrolled to further evaluate the clinical activity of FT819.

At the American Association for Cancer Research (AACR) Virtual 2020 Meeting, the Company presented preclinical data demonstrating FT819 is comprised of CD8 T cells with uniform 1XX CAR expression and complete elimination of endogenous TCR expression. Additionally, data from functional assessments showed FT819 has antigen-specific cytolytic activity in vitro against CD19-expressing leukemia and lymphoma cell lines that is comparable to that of healthy donor-derived CAR T cells, and persists and maintains tumor clearance in the bone marrow in an in vivo disseminated xenograft model of lymphoblastic leukemia.

Fate Therapeutics has an exclusive license for all human therapeutic use to U.S. Patent No. 10,370,452 pursuant to its license agreement with MSK1, which patent covers compositions and uses of effector T cells expressing a CAR, where such T cells are derived from a pluripotent stem cell including an iPSC. In addition to the patent rights licensed from MSK, the Company owns an extensive intellectual property portfolio that broadly covers compositions and methods for the genome editing of iPSCs using CRISPR and other nucleases, including the use of CRISPR to insert a CAR in the TRAC locus for endogenous transcriptional control.

1 Fate Therapeutics haslicensedintellectual propertyfrom MSK on which Dr. Sadelain is aninventor.As a result of the licensing arrangement, MSK has financial interests related to Fate Therapeutics.

About Fate Therapeutics iPSC Product Platform The 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 300 issued patents and 150 pending patent applications.

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 Statements This release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 including statements regarding the advancement of and plans related to the Company's product candidates and clinical studies, the Companys progress, plans and timelines for the clinical investigation of its product candidates, the therapeutic potential of the Companys product candidates including FT819, and the Companys clinical development strategy for FT819. 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 of difficulties or delay in the initiation of any planned clinical studies, or in the enrollment or evaluation of subjects in any ongoing or future 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 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), the risk that results observed in preclinical studies of FT819 may not be replicated in ongoing or future clinical trials or studies, and the risk that FT819 may not produce therapeutic benefits or may cause other unanticipated adverse effects. 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.

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

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Fate Therapeutics Announces FDA Clearance of IND Application for First-ever iPSC-derived CAR T-Cell Therapy - GlobeNewswire

Growing Focus on Product Innovation Likely to Impact the Growth of the Amniotic Fluid Stem Cell Therapy Market 2018 2026 – Jewish Life News

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Amniotic Fluid Stem Cell Therapy Market Table of Contents

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Growing Focus on Product Innovation Likely to Impact the Growth of the Amniotic Fluid Stem Cell Therapy Market 2018 2026 - Jewish Life News

Rheumatoid Arthritis Stem Cell Therapy Market Trends, Outlook and Opportunity Analysis 2018 to 2028 – Cole of Duty

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The report suggests that the global Rheumatoid Arthritis Stem Cell Therapy market is projected to reach a value of ~US$XX by the end of 2029 and grow at a CAGR of ~XX% through the forecast period (2019-2029). The key indicators such as the year-on-year (Y-o-Y) growth and CAGR growth of the Rheumatoid Arthritis Stem Cell Therapy market are discussed in detail in the presented report. This data is likely to provide readers an understanding of qualitative and quantitative growth prospects of the Rheumatoid Arthritis Stem Cell Therapy market over the considered assessment period.

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Rheumatoid Arthritis Stem Cell Therapy Market Trends, Outlook and Opportunity Analysis 2018 to 2028 - Cole of Duty

Ziopharm Oncology Announces Initiation of Phase 1 Trial Evaluating Rapid Personalized Manufacturing CAR-T Technology in Patients with Relapsed CD19+…

July 09, 2020 16:15 ET | Source: ZIOPHARM Oncology Inc

BOSTON, July 09, 2020 (GLOBE NEWSWIRE) -- Ziopharm Oncology, Inc. (Ziopharm or the Company) (Nasdaq:ZIOP), today announced the initiation of a phase 1 clinical trial to evaluate CD19-specific CAR-T, using its Rapid Personalized Manufacturing (RPM) technology, as an investigational treatment for patients with relapsed CD19+ leukemias and lymphomas. The trial is now open for enrollment at The University of Texas MD Anderson Cancer Center.

In this trial, the Company utilizes its non-viral Sleeping Beauty genetic engineering technology to infuse CAR-T the day after electroporation. Ziopharms RPM CD19-specific CAR-T therapy results from the stable, non-viral insertion of DNA into the genome of resting T cells to co-express the chimeric antigen receptor (CAR), membrane-bound IL-15 (mbIL15) and a safety switch.

We are pleased to expand the scope of our clinical development with MD Anderson, as we seek to evaluate our RPM technology using CD19-specific CAR-T cells, said Laurence Cooper, M.D., Ph.D., Chief Executive Officer of Ziopharm. RPM is a promising manufacturing solution, as T cells from the bloodstream are genetically reprogramed with DNA plasmids from the Sleeping Beauty system and then simply administered the next day.

Our CAR-T therapy can be administered at low cell doses, which may control cytokine release syndrome and is appealing for the treatment of patients including those with CD19-expressing malignancies that have relapsed after allogeneic bone marrow transplantation (BMT). There are limited effective treatment options for such patients as evidenced by the low rate of remission and poor long-term survival, Dr. Cooper added.

Up to 24 patients with advanced CD19+ leukemias and lymphomas who have relapsed after allogeneic BMT will be enrolled in this investigator-initiated trial (NCT03579888). The primary endpoint of the study is to determine the safety and maximum tolerated dose of donor-derived genetically modified CD19-specific T cells manufactured using the RPM process. An additional study is planned through Ziopharms joint venture with Eden BioCell to evaluate the RPM technology using patient-derived (autologous) CD19-specific CAR-T in Greater China.

Research reveals three-year survival for adults with CD19+ acute lymphoblastic leukemia after allogeneic BMT ranges from 30% to 65%.1 For patients with other CD19+ cancers, allogeneic BMT can provide three-year survival rates between 30% to 75%.1 Few patients experience a durable remission following allogeneic BMT, regardless of the treatment modality, with some having a median survival of only 2 to 3 months.2

About Ziopharm Oncology, Inc. Ziopharm is developing non-viral and cytokine-driven cell and gene therapies that weaponize the bodys immune system to treat the millions of people globally diagnosed with a solid tumor each year. With its multiplatform approach, Ziopharm is at the forefront of immuno-oncology with a goal to treat any type of solid tumor. Ziopharms pipeline is built for commercially scalable, cost effective T-cell receptor T-cell therapies based on its non-viral Sleeping Beauty gene transfer platform, a precisely controlled IL-12 gene therapy, and rapidly manufactured Sleeping Beauty-enabled CD19-specific CAR-T program. The Company has clinical and strategic collaborations with the National Cancer Institute, The University of Texas MD Anderson Cancer Center and Regeneron Pharmaceuticals. For more information, please visit http://www.ziopharm.com.

Forward-Looking Statements Disclaimer This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts, and in some cases can be identified by terms such as "may," "will," "could," "expects," "plans," "anticipates," and "believes." These statements include, but are not limited to, statements regarding the progress, design and timing of the Company's research and development programs, the potential benefits of the Companys therapies, and the Companys expectations regarding the number of patients in its clinical trials. Although Ziopharms management team believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Ziopharm, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include among other things, changes in our operating plans that may impact our cash expenditures, the uncertainties inherent in research and development, future clinical data and analysis, including whether any of Ziopharms product candidates will advance further in the preclinical research or clinical trial process, including receiving clearance from the U.S. Food and Drug Administration or equivalent foreign regulatory agencies to conduct clinical trials and whether and when, if at all, they will receive final approval from the U.S. FDA or equivalent foreign regulatory agencies and for which indication; the strength and enforceability of Ziopharms intellectual property rights; competition from other pharmaceutical and biotechnology companies as well as risk factors discussed or identified in the public filings with the Securities and Exchange Commission made by Ziopharm, including those risks and uncertainties listed in Ziopharms Quarterly Report on Form 10-Q filed by Ziopharm with the Securities and Exchange Commission. We are providing this information as of the date of this press release, and Ziopharm does not undertake any obligation to update or revise the information contained in this press release whether as a result of new information, future events or any other reason.

Investor Relations Contacts:Ziopharm Oncology: Chris Taylor VP, Investor Relations and Corporate Communications T: 617.502.1881 E: ctaylor@ziopharm.com

LifeSci Advisors: Mike Moyer Managing Director T: 617.308.4306 E: mmoyer@lifesciadvisors.com

Media Relations Contact:LifeSci Communications: Patrick Bursey T: 646.876.4932 E: pbursey@lifescicomms.com

1 D'Souza A, Fretham C. Current Uses and Outcomes of Hematopoietic Cell Transplantation (HCT): CIBMTR Summary Slides, 2018. Available at https://www.cibmtr.org

2 Keil F, Prinz E, Kalhs P, et al. Treatment of leukemic relapse after allogeneic stem cell transplantation with cytotoreductive chemotherapy and/or immunotherapy or second transplants. Leukemia 2001; 15:355-361.

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Ziopharm Oncology Announces Initiation of Phase 1 Trial Evaluating Rapid Personalized Manufacturing CAR-T Technology in Patients with Relapsed CD19+...

UK researchers use mini retinas to test gene therapy for RP – AOP

Scientists report promising results from a study that aims to develop a new approach to treating the most common inherited eye condition

Pixabay/Arek Socha

The study, which was published in Stem Cell Reports, involved using a viral vector to carry a replacement gene to malfunctioning cells in retinal organoids.

Following treatment, analysis revealed that the mini retinas had begun to produce a protein that is essential for vision.

Professor Mike Cheetham, from UCL, helped to develop the mini retinas that were used to test the gene therapy.

He highlighted that the mini retinas allow researchers to reproduce many different elements of inherited disease.

It makes it possible for us to study in detail why people go blind and try to find ways to prevent blindness. Its exciting that the gene therapy seems to be so effective for this form of retinitis pigmentosa, Professor Cheetham said.

The research was part-funded by Fight for Sight. The charitys chief executive, Sherine Krause, described the findings as incredibly promising.

Professor Cheetham and his teams work in collaboration with Trinity College Dublin represents a significant breakthrough for eye research and shows the importance of science to find new treatments for the prevailing causes of sight loss, she said.

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UK researchers use mini retinas to test gene therapy for RP - AOP

Stem Cell Therapy Market 2020-2029 Size, Share and Trends By Osiris Therapeutics, NuVasive, Chiesi Pharmaceuticals, JCR Pharmaceutical, Pharmicell -…

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Stem Cell Therapy Market Segmentation by Type:

Autologous Allogeneic

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Musculoskeletal Disorder Wounds & Injuries Cornea Cardiovascular Diseases Others

Market segment by Region/Country including: North America (United States, Canada and Mexico) Europe (Germany, UK, France, Italy, Russia and Spain etc.) Asia-Pacific (China, Japan, Korea, India, Australia and Southeast Asia etc.) South America Brazil, Argentina, Colombia and Chile etc.) Middle East & Africa (South Africa, Egypt, Nigeria and Saudi Arabia etc.)

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Stem Cell Therapy Market 2020-2029 Size, Share and Trends By Osiris Therapeutics, NuVasive, Chiesi Pharmaceuticals, JCR Pharmaceutical, Pharmicell -...