Stem Cell Clinic

Muscular dystrophy collaboration aims to correct muscle stem cells’ DNA – Harvard Office of Technology Development

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January 13, 2020

We expect that a satellite cell with the corrected DMD gene would quite quickly and continuously propagate the edited gene throughout the muscle tissue, said Prof. Amy Wagers, who leads the research. (Photo credit: Jon Chase/Harvard Staff Photographer.)

Cambridge, Mass. January 13, 2020 Harvard University stem cell researchers led by Amy Wagers, PhD, are embarking on a major study of Duchenne muscular dystrophy (DMD). Supported by research funding from Sarepta Therapeutics, under a multi-year collaboration agreement coordinated by Harvards Office of Technology Development (OTD), the project aims to use in-vivo genome editing, in mouse models of DMD, to fully and precisely restore the function of the dystrophin protein, which is crucial for proper muscular growth and development. Approaches validated by this work may point the way to an eventual therapeutic strategy to reverse DMD in humans.

Duchenne muscular dystrophy is a genetic disease caused by the lack of a protein called dystrophin that normally helps to support the structural integrity of muscle fibers, including those in the heart. Without the dystrophin protein, cells are weaker and degenerate more quickly. Over time, affected individuals boys, typically, as it is a recessive X-linked disorder lose their capacity to move independently.

Its really a devastating disease; it robs young boys of their capacity to be young boys, said Wagers, who is the Forst Family Professor of Stem Cell and Regenerative Biology, Co-Chair of the Department of Stem Cell & Regenerative Biology, and an Executive Committee Member of Harvard Stem Cell Institute. Though it is early days, Im hopeful that through this work we may identify and validate new avenues for therapy to completely rescue the proper expression and function of the dystrophin protein and regenerate healthy muscle tissue.

Researchers worldwide have pursued a variety of promising approaches such as cell and gene therapies, small-molecule therapies, and others to lessen or prevent the disease and improve patients quality of life.

The strategy pursued by the Wagers Lab at Harvard aims to fully correct the genetic template for dystrophin at its source, in the DNA of stem cells (satellite cells) that create and regenerate muscle cells. Combining cutting-edge CRISPR/Cas9 genome editing technologies with a deep knowledge of stem cell science and regenerative biology, this approach if successful might offer a permanent restoration of muscular function.

In skeletal muscle, muscle fibers are terminally post-mitotic, meaning they cannot divide and they cannot reproduce themselves, Wagers explains. If you lose muscle fibers, the only way to produce new muscle is from stem cells, specifically the satellite cells. The satellite cells are self-renewing, self-repairing, and ready to spring into action to create new muscle fibers. So we expect that a satellite cell with the corrected DMD gene would quite quickly and continuously propagate the edited gene throughout the muscle tissue.

At present, research conducted in mice has shown promising results. In June, the Wagers Lab published the results of editing stem cells in vivo, demonstrating that stem cell genes can be edited in living systems, not only in a dish. In that work, Wagers and her team delivered genome editing molecules to the cells using adeno-associated viruses (AAVs). Her lab has also successfully used gene editing in heart, muscle, and satellite cells to partially restore the function of the DMD gene that encodes dystrophin, by chopping out faulty sequences of code that are disrupting the proper reading frame.

The new stem-cell approach pursued in collaboration with Sarepta would build on these achievements and use more precise genome editing approaches, in animal models of DMD, to entirely replace genetic mutations in the DMD gene with correctly encoded sequences. The project will also explore alternate delivery methods and strategies to mitigate immune effects of in vivo genome editing.

This ambitious project will benefit greatly from the resources and insights of a company with deep clinical experience in the development of therapeutics for muscular dystrophy, said Vivian Berlin, Managing Director of Strategic Partnerships at Harvard OTD. Preclinical discoveries by Harvard researchers may open entirely new possibilities for lifesaving treatments in the long run, offering much-needed hope to patients and families in the future. Were grateful to be able to sustain the important momentum already established in Prof. Wagers lab, through this collaboration.

As we work to bring forward new treatments for patients with DMD, Sarepta is excited to support Prof. Wagers and her lab to accelerate the development of a gene editing approach, which has shown significant potential in early studies, said Louise Rodino-Klapac, Sareptas Senior Vice President of Gene Therapy. This multi-year collaboration is part of Sareptas broader commitment to pursuing all therapeutic modalities and advancing our scientific understanding of gene editing in order to maximize the potential of this approach to help patients.

Under the terms of the agreement between Harvard and Sarepta, the company will have the exclusive option to license any arising intellectual property for the purpose of developing products to prevent and treat human disease. As with any research agreement facilitated by OTD, the right of academic and other not-for-profit researchers to use the technology in further scholarly work is preserved.

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Muscular dystrophy collaboration aims to correct muscle stem cells' DNA - Harvard Office of Technology Development

AstroRx Increased Ability to Perform Daily Functions in ALS Patients – ALS News Today

Amyotrophic lateral sclerosis (ALS) patients receiving the lowest dose of Kadimastems cell therapy candidate,AstroRx, experienced a significant reduction in disease progression in the three or four months after treatment, updated findings from the companys Phase 1/2 clinical trial show.

In subsequent months, however, the disease deteriorated at similar rates as before treatment, suggesting that repeat dosing or higher doses both being tested in the ongoing trial may increase AstroRxs effectiveness.

No serious treatment-related adverse events or dose-limiting toxicities were reported in the first group of patients at least six months after treatment, suggesting that the lowest dose (100 million cells) is safe and well-tolerated.

These results on a small subset of patients of the first experimental group treated with 100 million AstroRx cells are encouraging, as the treatment seems to be safe, Marc Gotkine, MD, department of neurology at Hadassah Medical Center, Jerusalem, said in a press release.

After completion of 6 months follow up period, initial data analysis presented to us appears to demonstrate a transient efficacy benefit that lasted for 34 months post treatment, said Gotkine, who also is the principal investigator of the trial. The trial is now continuing with higher doses and repeated injections.

AstroRx is a cell therapy made of healthy, mature astrocytes, which are star-shaped cells of the central nervous system that normally maintain a healthy brain environment but are abnormal in ALS patients, contributing to disease progression.

The off-the-shelf therapy is derived from human embryonic stem cells and is expected to compensate for diseased astrocytes, preventing the loss of motor nerve cells when injected into a patients spinal fluid.

Studies in animals showed that AstroRx is safe and can delay ALS onset, maintain muscle function and increase survival. This led the biotech company in Israelto opena single-site Phase 1/2a clinical trial (NCT03482050) evaluating escalating doses of AstroRx in ALS patients.

The open-label study is being conducted at theHadassah Ein-Kerem Medical Centerand is expected to enroll 21 people withearly stage disease. Recruitmentis ongoing.

The main goal is to determine safety and tolerability of the therapy. Secondary measures include changes in patients ALS Functional Rating Scale revised(ALSFRS-R) scores, respiratory muscle strength, hand grip strength, limb muscle strength, and quality of life.

The company shared data from the first five patients included in the trial, all of whom received a single infusion of 100 million cells the lowest dose of AstroRx tested and were followed for at least six months after treatment.

After confirming the treatments safety, researchers assessed its efficacy and the duration of therapeutic effects measured through changes in ALSFRS-R scores, which cover speech, swallowing, dressing and hygiene, among other daily functions.

In the three months preceding AstroRxs treatment, patients ALSFRS-R score dropped by 0.87 points per month, on average, a similar decline in functional status as described in prior ALS studies.

In the three months after treatment, however, ALSFRS-R raised by an average of 0.26 points per month, suggesting that patients ability to perform daily activities increased in this period.

Researchers also examined the average ALSFRS-R change at four, five, and six months after treatment. While the rate of disease progression was still significantly lower in the first four months than before treatment (with an average decline of 0.32 points per month), at five and six months it was similar to the pre-treatment period.

That indicates this dose of AstroRx is effective only for a period of three or four months, after which patients see their disease deteriorate at a similar rate as before treatment.

We are very encouraged by the final cohort A results, demonstrating AstroRx provides a meaningful clinical benefit in terms of the ALSFRS-R rating scale, said Rami Epstein, CEO of Kadimastem. We look forward to continue our study, assessing the potential long-term benefits of a higher dose regimen as well as repeated administrations of our breakthrough cell therapy.

The trial is now assessing a higher dose of the therapy (250 million cells) in another group of five patients, all of whom have been enrolled. Kadimastem has recruited the first patient into a third group that will receive two AstroRx injections of 100 million cells, separated by two to three months months.

Results from the second and third groups are expected by August and the first half of 2021, respectively. Pending positive safety and effectiveness results, a fourth group will receive two injections of 250million cells.

Since in cohort A the clinical response was clearly demonstrated to last for at least 34 months following cell injection, cohort B will include a higher dose of cells, and cohort C will include repeated injections with approximately 3-month intervals, said Michel Revel, MD, PhD, founder and chief scientific officer of Kadimastem. Both cohort[s] B and C hold a potential promise for a prolonged response. The positive safety profile also allows us to test AstroRx in other neurodegenerative diseases, he said.

Ins holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she specialized in blood vessel biology, blood stem cells, and cancer. Before that, she studied Cell and Molecular Biology at Universidade Nova de Lisboa and worked as a research fellow at Faculdade de Cincias e Tecnologias and Instituto Gulbenkian de Cincia. Ins currently works as a Managing Science Editor, striving to deliver the latest scientific advances to patient communities in a clear and accurate manner.

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AstroRx Increased Ability to Perform Daily Functions in ALS Patients - ALS News Today

Fate Therapeutics Announces Expansion of FT516 Clinical Investigation and Publication of Preclinical Data in the Journal Blood – Yahoo Finance

FT516 IND Application Cleared by FDA for Advanced Solid Tumors in Combination with PDL1-, EGFR- and HER2-targeting Therapeutic Antibodies

Published Preclinical Data Demonstrate iPSC-derived NK Cells Engineered with High-affinity, Non-cleavable CD16 Enhance the Efficacy of Antibody Therapy

SAN DIEGO, Jan. 13, 2020 (GLOBE NEWSWIRE) -- Fate Therapeutics, Inc. (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 allowed its second Investigational New Drug (IND) application for FT516, the Companys off-the-shelf natural killer (NK) cell product candidate derived from a clonal master induced pluripotent stem cell (iPSC) line engineered to express a novel CD16 Fc receptor. This is the Companys fourth IND from its proprietary iPSC product platform cleared by the FDA, and enables the clinical investigation of FT516 in combination with monoclonal antibody (mAb) therapy across a broad range of solid tumors.

While monoclonal antibodies are proven therapeutic agents that are often used early in the treatment of many cancers, the functional status of the patients NK cells has been shown to play an important role in mediating clinical activity and prolonging survival, said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. In particular, stable expression of the NK cell activating receptor CD16, and its binding affinity to therapeutic antibodies, are critical to promoting antibody-dependent cellular cytotoxicity. Our first-of-kind, off-the-shelf approach with FT516 enables administration of multiple doses of CD16-engineered NK cells, and we are excited to investigate the potential of FT516 to augment the clinical efficacy of monoclonal antibody therapy in the setting of solid tumors.

FT516 expresses a novel high-affinity, non-cleavable variant of CD16 (hnCD16) that enhances its binding to therapeutic antibodies and prevents its down-regulation, which can significantly inhibit anti-tumor activity. A publication by scientists from the Company, the University of Minnesota, and the University of California, San Diego in the journal Blood (https://doi.org/10.1182/blood.2019000621), entitled Pluripotent stem cell-derived NK cells with high-affinity non-cleavable CD16a mediate improved anti-tumor activity, highlights preclinical proof-of-concept data for FT516.

In the published studies, iPSC-derived NK cells expressing hnCD16 were shown to have superior therapeutic properties in vitro, including maintenance of CD16 expression and increased levels of cytokine production upon activation, compared to peripheral blood NK cells sourced from healthy donors. In an in vivo systemic tumor model of human lymphoma, treatment with iPSC-derived hnCD16 NK cells plus anti-CD20 mAb resulted in a significant improvement in survival (median survival exceeding 100 days) compared to treatment with anti-CD20 mAb alone or in combination with peripheral blood NK cells sourced from healthy donors (each of which showed median survival of 35 days). Additionally, iPSC-derived hnCD16 NK cells plus anti-HER2 mAb also conveyed a survival benefit in a xenograft model of SKOV-3 ovarian carcinoma.

FT516 is the first-ever cell therapy in the world derived from a genetically engineered pluripotent stem cell cleared for clinical testing. The Company intends to initiate clinical investigation of FT516 in combination with tumor-target antibody therapy in solid tumors later this year. The Company is currently conducting an open-label, multi-dose Phase 1 clinical trial of FT516 as a monotherapy for the treatment of acute myeloid leukemia and in combination with CD20-directed mAbs for the treatment of advanced B-cell lymphoma.

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

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

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

Forward-Looking StatementsThis release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 including statements regarding the safety and therapeutic potential of the Companys NK cell product candidates, including FT516, its ongoing and planned clinical studies, and the expected clinical development plans for FT516. These and any other forward-looking statements in this release are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that the Company may cease or delay planned development and clinical trials of any of its product candidates for a variety of reasons (including any delay in enrolling patients in current and planned clinical trials, requirements that may be imposed by regulatory authorities on the conduct of clinical trials or to support regulatory approval, difficulties in manufacturing or supplying the Companys product candidates for clinical testing, or the occurrence of any adverse events or other negative results that may be observed during development), the risk that results observed in preclinical studies of its product candidates, including FT516, may not be replicated in ongoing or future clinical trials or studies, and the risk that its product candidates 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 TartagliaStern Investor Relations, Inc.212.362.1200christina@sternir.com

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Fate Therapeutics Announces Expansion of FT516 Clinical Investigation and Publication of Preclinical Data in the Journal Blood - Yahoo Finance

Police Arrest 3 in Illegal Stem Cell Therapy Allegations – Tempo.co English

TEMPO.CO, Jakarta -Three people have been arrested and named as suspects in the case of illegal stem cell therapy practice in Kemang. The Jakarta Metro Police detained doctors and owners of the clinic after they raided the place Saturday afternoon.

"Previously, three people with the initials Y, O, and L were interrogated. They have been named as suspects are now detained at the Metro Police Headquarters," Jakarta Police PR chief Comr. Yusri Yunus said on Monday, January 13, 2020.

Yusri declined to further disclose the progress of the Police's investigation, and said that the Police will hold a press conference on Tuesday, January 14, 2020.

The Police recently sealed a clinic at Ruko Bellepoint in Kemang, South Jakarta, after it was proven to give stem cell injections to patients illegally.

"The clinic has been operating in Indonesia for three years," Jakarta Metro Police's director of general criminal investigation, Sr. Comr. Suyudi Ario Seto said.

Police investigators named the clinic's manager YW (46), marketing manager LJ (47), and Dr. OH as suspects.

During the bust, officers also confiscated a number of items of evidence such as unlicensed stem cells products from Japan, IV tubes, syringes, antiseptic tools, as well as documents of patients' registration.

The illegal stem cell therapy practice is against a number of articles in the criminal code as well as the Doctor of Law Practice of 2004 (Law no.29/2004) as well as a number of other provisions.

ANTARA

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Police Arrest 3 in Illegal Stem Cell Therapy Allegations - Tempo.co English

Stem Cell Therapy Market Competitive Analysis 2019-2027 – News ZMR

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Stem Cell Therapy Market Competitive Analysis 2019-2027 - News ZMR

First ever progressive MS treatment approved for use by NHS in Scotland – The Scotsman

A leading health charity has welcomed the watershed decision to approve the first ever drug used in the treatment of progressive multiple sclerosis (MS) in Scotland.

Ocrelizumab (Ocrevus) has been given the green light by the Scottish Medicines Consortium (SMC) for treating primary progressive MS where the condition is still early in terms of duration and disability and shows evidence of inflammation.

It becomes the first drug to be made available on the NHS in Scotland for primary progressive MS. There are currently 13 treatments available for people with the relapsing form of the condition.

READ MORE: https://www.scotsman.com/health/groundbreaking-stem-cell-therapy-for-multiple-sclerosis-recommended-for-use-in-scotland-1-5035721This news follows an SMC decision to make ocrelizumab available on NHS Scotland for people with relapsing MS in December 2018 as well as the approval, in May 2019, for its use for people with primary progressive MS in England.

More than 11,000 people in Scotland have MS and around 65 each year are diagnosed with the primary progressive form. Ocrelizumab is the first and only treatment that can slow disability progression in this type of MS, where symptoms gradually worsen over time. It is licensed for early primary progressive MS, which is defined by how long someone has lived with MS symptoms, their level of disability, and MRI scans showing inflammatory activity.

Karine Mather was diagnosed with primary progressive MS six years ago and she, and her wife Sarah, have seen their lives impacted hugely since then. They reflected on what this decision will mean for people in a similar situation in the future.

She said: This is great news for the MS community as people diagnosed with early primary progressive MS in Scotland will now be able to access a treatment for the first time.

Primary Progressive MS has had a massive impact on my life and on the lives of my wife and family.

In the space of five years I went from walking with a slight limp and working full-time to using a powerchair, unable to work and needing round the clock care from my wife who gave up her full-time job.

This medication will slow the progression of MS offering people newly diagnosed a treatment, enabling them to continue working and living a full life."

Sarah said: Since Karine was diagnosed with primary progressive MS we have gone from a working household with two full time incomes to her being unable to work and myself only able to work 10 hours per week due to my caring responsibilities.

Access to ocrelizumab could help people continue to live their lives and help slow progression of the disabilities associated with MS so its wonderful news that others could now benefit from this treatment.

READ MORE: https://www.scotsman.com/health/drugs-trial-offers-new-hope-for-edinburgh-ms-sufferer-1-4855730MS Society Scotland hailed the landmark news and the positive effect it could have for people across the country.

Morna Simpkins, Director for MS Society Scotland said: This is great news and a hugely important development for people diagnosed with primary progressive MS in Scotland.

We want every one of the 11,000 people in Scotland living with MS to have access to the right treatment at the right time and this decision takes us closer than ever to that goal.

Right now, however, there isnt enough evidence to show ocrelizumab can work for everyone, and we know the limited scope of this announcement will be disappointing for those who still dont have any options. Were driving research to find more and better treatments, and calling for drug trials to more fully address the needs of everyone with MS, until the day we are able to stop it in its tracks.

Research has got us to a critical point, and we can see a future where nobody needs to worry about MS getting worse. Our Stop MS appeal is aiming to raise 100 Million over 10 years to make that a reality and build on the treatments, like ocrelizumab, that are available.

Ocrelizumab works by attaching itself to a protein (called CD20) on the surface of a type of white blood cell (called B cells) that attacks the protective coating around the nerves causing inflammation and damage to the nerve fibres. As a result ocrelizumab can block their activity and reduce the symptoms of MS.

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First ever progressive MS treatment approved for use by NHS in Scotland - The Scotsman

Editas Medicine and Sandhill Therapeutics, Inc. Announce Collaboration to Develop Engineered Cell Medicines to Treat Cancer | More News | News…

DetailsCategory: More NewsPublished on Monday, 13 January 2020 17:41Hits: 228

CAMBRIDGE, MA and DALLAS, TX, USA I January 13, 2020 I Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, and Sandhill Therapeutics, Inc., a cellular immuno-oncology company, announced a strategic research collaboration, license, and option agreement to combine their respective genome editing and cell therapy technologies to discover, develop, and manufacture allogeneic engineered natural killer (NK) cells and non-alpha beta T cell medicines for the treatment of cancer.

This collaboration brings together Editas Medicines leading genome editing technology and Sandhills BINATE product process, a novel universal donor technology to extract, isolate, and expand NK cells and non-alpha beta T cells, to develop novel medicines for the treatment of solid tumor cancers.

We are excited to work with Sandhill, combining CRISPR-based genome editing with BINATE cells to accelerate the development of numerous, transformative medicines for people with cancer and improve patient outcomes, said Charles Albright, Ph.D., Executive Vice President and Chief Scientific Officer, Editas Medicine. We continue to increase our commitment to oncology, and we believe our portfolio of multiple immune system cell types, including T cells, NK cells, and induced pluripotent stem cells (iPSCs), will be effective in making the next generation of allogeneic medicines to fight many common cancers.

The team at Editas Medicine has one of the most innovative technology platforms, and we look forward to combining our technologies to create new medicines for the treatment of cancer. Together, we are dedicated to transforming cellular immuno-oncology and developing new therapies, said Annemarie Moseley, M.D., Ph.D., Chief Executive Officer, Sandhill Therapeutics, Inc.

Under the terms of the agreement, Editas Medicine obtains an exclusive license to Sandhills technology to research, develop and commercialize immuno-oncology engineered cell medicines for solid tumors originating within a given area of the body and an option to expand such license to two additional areas. In return, Sandhill will receive an upfront payment, development and sales-based milestone payments, and royalties on sales of resulting Editas products.

RBC Capital Markets acted as exclusive financial advisor to Sandhill for the transaction.

About Editas MedicineAs a leading genome editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas9 and CRISPR/Cpf1 (also known as Cas12a) genome editing systems into a robust pipeline of treatments for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases. For the latest information and scientific presentations, please visit http://www.editasmedicine.com.

About Sandhill Therapeutics, Inc.Sandhill Therapeutics is a privately held, development stage cellular immunotherapy company dedicated to improving the lives of children and adults with cancer. Sandhills BINATE leverages dual innate cell synergy, resulting in a highly activated, readily available, universal off-the-shelf treatment for both solid tumors and blood cancers. Sandhills activated innate cell immunotherapy is generated by a cost-effective, feeder-free campaign manufacturing process. For more information, visitwww.sandhilltx.com.

SOURCE: Editas Medicine

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Editas Medicine and Sandhill Therapeutics, Inc. Announce Collaboration to Develop Engineered Cell Medicines to Treat Cancer | More News | News...

Stemline Therapeutics Announces Preliminary 2019 Net Revenues for ELZONRIS (tagraxofusp) and Highlights Commercial and Clinical Growth Drivers – Yahoo…

NEW YORK, Jan. 13, 2020 (GLOBE NEWSWIRE) -- Stemline Therapeutics, Inc. (STML), a commercial-stage biopharmaceutical company focused on the development and commercialization of novel oncology therapeutics, today announced preliminary net revenues for 2019, as well as outlined key BPDCN market successes and upcoming commercial and clinical milestones.

Unaudited preliminary 2019 results include:

The above financial information is based on preliminary unaudited information, is subject to adjustment, and does not present all information necessary for an understanding of the Companys full-year and fourth quarter financial results for 2019. Stemline expects to report complete audited 2019 financial results on or before March 16, 2020.

Robert Francomano, Chief Commercial Officer of Stemline, stated, We are very pleased with the solid uptake seen in the first year of the ELZONRIS launch, as we continue to successfully create, penetrate and grow a new market in BPDCN. Given the orphan nature and unique features of this disease, we believe patient starts were subject to significant quarterly variance a phenomena that will likely continue throughout 2020. We are actively implementing a host of tactics to expand and further penetrate this emerging market.

Ivan Bergstein, CEO of Stemline, commented, 2019 was a transformational year for Stemline as we launched ELZONRIS, the first and only CD123 targeted agent and first agent ever approved for patients with BPDCN. We continue to pursue growth opportunities not only in BPDCN but also in a number of malignancies where targeting CD123 could provide therapeutic benefit. We look forward to data readouts in CMML, MF, and AML, including in patient subsets with high CD123, later this year and on into next year. Given our continued commercial and clinical progress, we look forward to a productive 2020 and beyond.

Corporate Highlights and Key Commercial and Clinical Milestones

BPDCN

Chronic Myelomonocytic Leukemia (CMML)

Myelofibrosis (MF)

Acute Myeloid Leukemia (AML)

About ELZONRISELZONRIS(tagraxofusp), a CD123-directed cytotoxin, is approved by the U.S. Food and Drug Administration (FDA) and commercially available in the U.S. for the treatment of adult and pediatric patients, two years or older, with blastic plasmacytoid dendritic cell neoplasm (BPDCN). For full prescribing information in the U.S., visit http://www.ELZONRIS.com. In Europe, a marketing authorization application (MAA) is under review by the European Medicines Agency (EMA). ELZONRIS is also being evaluated in additional clinical trials in other indications including chronic myelomonocytic leukemia (CMML), myelofibrosis (MF), and acute myeloid leukemia (AML).

About BPDCN BPDCN is an aggressive hematologic malignancy with historically poor outcomes and an area of unmet medical need. BPDCN typically presents in the bone marrow and/or skin and may also involve lymph nodes and viscera. The BPDCN cell of origin is the plasmacytoid dendritic cell (pDC) precursor. The diagnosis of BPDCN is based on the immunophenotypic diagnostic triad of CD123, CD4, and CD56, as well as other markers. For more information, please visit the BPDCN disease awareness website at http://www.bpdcninfo.com.

About CD123CD123 is a cell surface target expressed on a wide range of myeloid tumors including blastic plasmacytoid dendritic cell neoplasm (BPDCN), certain myeloproliferative neoplasms (MPNs) including chronic myelomonocytic leukemia (CMML) and myelofibrosis (MF), acute myeloid leukemia (AML) (and potentially enriched in certain AML subsets), myelodysplastic syndrome (MDS), and chronic myeloid leukemia (CML). CD123 has also been reported on certain lymphoid malignancies including multiple myeloma (MM), acute lymphoid leukemia (ALL), hairy cell leukemia (HCL), Hodgkins lymphoma (HL), and certain Non-Hodgkins lymphomas (NHL). In addition, CD123 has been detected on some solid tumors as well as autoimmune disorders including cutaneous lupus and scleroderma.

About Stemline Therapeutics Stemline Therapeutics, Inc. is a commercial-stage biopharmaceutical company focused on the development and commercialization of novel oncology therapeutics. ELZONRIS(tagraxofusp), a targeted therapy directed to CD123, is FDA-approved and commercially available in the U.S. for the treatment of adult and pediatric patients, two years and older, with blastic plasmacytoid dendritic cell neoplasm (BPDCN). In Europe, a marketing authorization application (MAA) is under review by the European Medicines Agency (EMA). ELZONRIS is also being evaluated in clinical trials in additional indications including chronic myelomonocytic leukemia (CMML), myelofibrosis (MF) and acute myeloid leukemia (AML). Additional pipeline candidates include: felezonexor (SL-801) (XPO1 inhibitor; Phase 1 in advanced solid tumor patients ongoing), SL-1001 (novel RET kinase inhibitor, IND-enabling studies ongoing), SL-701 (immunotherapeutic; Phase 2 in glioblastoma patients completed), and SL-901 (novel kinase inhibitor; prior abbreviated European Phase 1, IND-enabling studies ongoing). For more information, please visit the companys website at http://www.stemline.com.

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Forward-Looking StatementsSome of the statements included in this press release may be forward-looking statements that involve a number of risks and uncertainties. For those statements, we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995. The factors that could cause our actual results to differ materially include: the risk that our actual revenue for the fourth quarter and year endedDecember 31, 2019may differ materially from our estimated results for these periods as a result of the completion of year-end closing procedures or the audit of our financial statements; the success of our U.S. launch and commercialization; the success of our MAA submission to the EMA and potential launch in Europe; the success and timing of our clinical trials and preclinical studies for our product and product candidates, including ELZONRIS in additional indications and our other pipeline candidates, including site initiation, institutional review board approval, scientific review committee approval, patient accrual, safety, tolerability and efficacy data observed, and input from regulatory authorities including the risk that the FDA, EMA, or other ex-U.S. national drug authority ultimately does not agree with our data, find our data supportive of approval, or approve any of our product candidates; the possibility that results of clinical trials are not predictive of safety and efficacy results of our product candidates in broader patient populations or of our products if approved; our plans to develop and commercialize our product candidates, including, but not limited to delays in arranging satisfactory manufacturing capabilities and establishing commercial infrastructure for ELZONRIS; product efficacy or safety concerns resulting in product recalls or regulatory action; the risk that estimates regarding the number of patients with the diseases that our product and product candidates may treat are inaccurate; inadequate market penetration of our products; our products not gaining acceptance among patients (and providers or third party payors) for certain indications (due to cost or otherwise); the risk that third party payors (including governmental agencies) will not reimburse for the use of ELZONRIS at acceptable rates or at all; the companys ability to produce, maintain or increase sales of ELZONRIS; the companys ability to develop and/or commercialize ELZONRIS; the adequacy of our pharmacovigilance and drug safety reporting processes; our available cash and investments; our ability to obtain and maintain intellectual property protection for our product and product candidates; delays, interruptions, or failures in the manufacture and supply of our product and product candidates; the performance of third-party businesses, including, but not limited to, manufacturers, clinical research organizations, clinical trial sponsors and clinical trial investigators; and other risk factors identified from time to time in our reports filed with the SEC. Any forward-looking statements set forth in this press release speak only as of the date of this press release. We do not intend to update any of these forward-looking statements to reflect events or circumstances that occur after the date hereof.

Contact: Investor RelationsStemline Therapeutics, Inc.750 Lexington AvenueEleventh FloorNew York, NY 10022Tel: 646-502-2307Email: investorrelations@stemline.com

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Stemline Therapeutics Announces Preliminary 2019 Net Revenues for ELZONRIS (tagraxofusp) and Highlights Commercial and Clinical Growth Drivers - Yahoo...

The potential use of CRISPR to treat disease is gaining momentum – BioNews

13 January 2020

Promising results from clinical trials give hope for using CRISPR/Cas9 genome editing to treat various heritable diseases and cancer in humans.

It has been seven years since the discovery that the CRISPR/Cas9 defence system, used by microbes to destroy viruses, could be re-engineered to edit the human genome. Since then researchers have carried out an array of experiments to explore potential applications.

Biophysist Dr He Jiankui sparked global controversy concerning the ethics of genome editing when he used CRISPR to genetically modify embryos, resulting in the birth of the first genome-edited babies (see BioNews 977).

Yet researchers worldwide have at the same time been investigating the use of CRISPR for non-heritable changes, modifying the genes in non-embryonic cells to treat a wide range of diseases.

'There's been a lot of appropriate caution in applying this to treating people, but I think we're starting to see some of the results of that work,' said Dr Edward Stadtmauer, a haematologist at the University of Pennsylvania, Philadelphia.

Over a dozen new clinical trials testing CRISPRtherapy on diseases such as cancer, HIV and sickle cell anaemia were listed on the clinicaltrials.gov database last year. One trial in its early stages used CRISPR to treat sickle cell anaemia and beta-thalassaemia, both genetic blood disorders that result in the production of an abnormal form of the oxygen-carrying protein, haemoglobin.

Two patients with these disorders were treated by CRISPR Therapeutics in Cambridge, Massachusetts, and Vertex Pharmaceuticals in Boston, Massachusetts, using CRISPR to inactivate a gene that switches off the production of an alternative form of haemoglobin. Preliminary results of the study suggest that this therapy improved some of the symptoms but the participants will need to be followed for a longer period to be sure.

Results from two other trials, one in which genome-edited blood cells were transplanted into a man to treat HIV infection, and the other in which they were transplanted into three people to treat some forms of cancer, were less successful. In both cases, the transplanted cells flourished in the bone marrow of recipients, without any serious safety concerns, but did not produce a clear medical benefit. The study has been placed on hold while researchers explore ways to boost that percentage, says Hongkui Deng, a stem-cell researcher at Peking University, Beijing, China and a lead author of the work.

Other researchers are trying to move beyond editing cells in vitro. In July 2019 a clinical trial was launched to treat Leber congenital amaurosis 10 (LCA10), a rare genetic disease that causes blindness. The trial, launched by two pharmaceutical companies, Editas Medicine in Cambridge, Massachusetts, and Allergan in Dublin, Ireland, will be the first trial that uses CRISPR to edit cells inside of the body. The researchers are testing AGN-151587 (EDIT-101), which is a novel CRISPR treatment delivered via adeno-associated virus (AAV) directly to the eye's light-sensing photoreceptor cells to remove the mutation that causes LCA10.

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The potential use of CRISPR to treat disease is gaining momentum - BioNews