ST-501

Pictured: Novartis Headquarters/Courtesy of Getty Images Sangamo Therapeutics lost two powerhouse partners Friday after it reported that both Novartis and Biogen discontinued their partnerships studying gene regulation therapies in the neurology space. Biogen and Novartis terminated the agreements following a strategic review, according to Sangamo’s SEC filing posted Mar. 13. Both collaborations will formally end in June 2023. Sangamo will conduct a broader strategic review of its pre-clinical development assets and investigate alternatives for the development programs left behind by Biogen and Novartis. Depending on the result of its review, Sangamo could continue these programs internally or look for collaborators. Neither Novartis or Biogen has reponded to BioSpace's request for comment. As of year-end 2022, Sangamo had $307.5 million in cash, cash equivalents and marketable securities, down considerably from its $464.7 million balance in 2021. Still, the company reported that its funds would be enough to support operations for at least 12 months. Sangamo representatives said the termination of the Novartis and Biogen partnerships shouldn’t affect its financial standing. Zinc Finger Partnerships Sangamo inked its partnership with Biogen in February 2020. For $350 million upfront and the promise of up to $2.37 billion in milestone payments, Biogen gained access to Sangamo’s genome regulation technology to advance candidates for up to 12 neurological and neuromuscular conditions. Biogen ultimately selected four candidates: ST-501 for tauopathies, ST-502 for synucleinopathies including Parkinson’s disease, a third product for type 1 myotonic dystrophy and a fourth candidate for an undisclosed neurological disease gene target. In July 2020, Sangamo signed to discover and develop gene regulation therapies for three neurodevelopmental conditions, including autism spectrum disorder. The Novartis deal was valued at $75 million upfront and up to $720 million in developmental and commercial milestones. Beyond the effective termination dates, Sangamo will no longer be eligible to receive milestones and royalties from its partners, nor will Biogen or Novartis be obligated to reimburse research activities related to their respective neuro programs. At the core of both the Biogen and Novartis partnerships was Sangamo’s proprietary zinc finger technology, which combines a DNA-binding domain with various functional domains like nucleases or transcription factors. Sangamo has a deep library with thousands of zinc fingers, allowing the company to cater the design of each candidate to a specific therapeutic use.

Dublin, May 26, 2021 (GLOBE NEWSWIRE) -- The "Gene and Cell Therapies Targeting CNS Disorders - Market Insights and Market Forecast - 2026" report has been added to ResearchAndMarkets.com's offering. This 'Gene and Cell Therapies targeting CNS Disorders - Market Insights and Market Forecast - 2026' report delivers an in-depth understanding of the market trends of Gene and Cell Therapies targeting CNS Disorders in in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan. The Gene and Cell Therapies targeting CNS Disorders market report provides current treatment practices, emerging drugs, market share of the individual therapies, current and forecasted Gene and Cell Therapies targeting CNS Disorders market size from 2018 to 2026, in the 7MM. The report also covers the current Gene and Cell Therapies targeting CNS Disorders treatment practice, market drivers, market barriers, and unmet medical needs to curate the best opportunities and assesses the underlying market potential. Gene and Cell Therapies targeting CNS Disorders emerging drugs NurOwn (BrainStorm Cell Therapeutics) is is mesenchymal stromal stem cells based therapy secreting neurotrophic factors (MSC-NTF) cells. It is designed to target disease pathways in neurodegenerative disorders by effectively delivering NTFs directly to the site of damage. MSC-NTF cells are generated using autologous, mesenchymal stem cells (MSCs) that have been extracted from bone marrow, expanded and differentiated ex-vivo. The therapy is currently under clinical evaluation in phase III for the treatment of Amyotrophic lateral sclerosis (ALS) and in phase II for treatment of Multiple Sclerosis, Chronic Progressive. Neuro-Cells (Neuroplast) is an autologous cell preparation that aims to decrease inflammation and reduce cell death. Neuro- Cells is produced in a certified and patented closed system manufacturing process from bone marrow and is a registered Advanced Therapy Medicinal Product (ATMP). Based on these mechanisms, Neuroplast focuses on developing a stem cell therapy, which can inhibit the inflammatory processes following damaged/dying neural tissue cells and sees its Neuro-Cells as a disease-modifying therapy (DMT). Currently it is being evaluated in phase I and phase II/III clinical studies in treatment of Spinal Chord Injuries (SCI). Gene and Cell Therapies targeting CNS Disorders Drugs Uptake This section focuses on the rate of uptake of the potential drugs recently launched or expected to get launched in the market during the study period 2018-2026. The analysis covers Gene and Cell Therapies targeting CNS Disorders market uptake by drugs, patient uptake by therapies, and sales of each drug. This helps in understanding the drugs with the most rapid uptake, reasons behind the maximal use of new drugs, and allow the comparison of the drugs based on market share and size, which again will be useful in investigating factors important in the market uptake and in making financial and regulatory decisions. Gene and Cell Therapies targeting CNS Disorders Pipeline Development Activities The report provides insights into different therapeutic candidates in Phase III, Phase II/III, Phase II, and Phase I/II stage. It also analyses Gene and Cell Therapies targeting CNS Disorders' key players involved in developing targeted therapeutics. Pipeline Development Activities The report covers the detailed information of collaborations, acquisition, and merger, licensing, and patent details for Gene and Cell Therapies targeting CNS Disorders emerging therapies. Key Topics Covered: 1. Key Insights2. Report Introduction3. Executive Summary4. Gene and Cell Therapies targeting CNS disorders: Overview4.1. Introduction to CNS Disorders4.2. Role of Cell and Gene Therapy in CNS Disorders4.3. Multiple Sclerosis4.4. Parkinson's disease4.5. Spinal cord injury4.6. Amyotrophic Lateral Sclerosis4.7. Huntington's disease4.8. Alzheimer's disease4.9. Spinal Muscular Atrophy5. Landscape Assessment5.1. Regulatory Scenario of Cell and Gene Therapies in the 7MM5.2. United States5.3. The European Union5.3.1. United Kingdom5.3.2. Germany5.3.3. France5.3.4. Italy5.3.5. Spain5.4. Japan5.5. Reimbursement Scenario of Cell and Gene Therapies in the 7MM5.5.1. United States5.5.2. United Kingdom5.5.3. Germany5.5.4. France5.5.5. Italy5.5.6. Spain5.5.7. Japan5.6. Mergers and Acquisitions5.7. Industry News and Policies by Regions5.7.1. Industry News5.7.2. Policies and Government Initiatives for Cell and Gene Therapies6. Marketed Products6.1. Zolgensma: Novartis7. Emerging Therapies for Amyotrophic Lateral Sclerosis7.1. NurOwn: BrainStorm Cell Therapeutics7.2. Engensis: Helixmith7.3. HYNR-CS inj: Corestem7.4. Q Cells: Q therapeutics7.5. VM202: Helixmith7.6. RAPA-501 Autologous T cells: Rapa Therapeutics8. Emerging Therapy for Multiple sclerosis8.1. NurOwn: BrainStorm Cell Therapeutics8.1.1. Product Description8.1.2. Product Developmental Activities9. Emerging Therapy for Spinal Cord Injury9.1. Neuro-Cells: Neuroplast9.2. Umbilical Cord Blood Mononuclear Cell: StemCyte9.3. FAB117-HC: Ferrer Internacional9.4. Human spinal cord stem cells: Neuralstem9.5. FAB117-HC: Ferrer Internacional10. Emerging therapies for Alzehimer10.1. itMSCs: Stemedica Cell Technologies10.2. AAV-hTERT: Libella Gene Therapeutics10.3. ST-501: Sangamo Therapeutics11. Emerging Therapies for Parkinson Disease11.1. VY-AADC: Hoffmann-La Roche11.2. Lomecel-B: Longeveron11.3. OXB-102: Sio Gene Therapies11.4. PR001: Prevail Therapeutics (Eli Lilly and Company)11.5. Neural Stem Cell-Derived Neurons: NeuroGeneration11.6. AAV2-GDNF: Brain Neurotherapy Bio (AskBio)12. Emerging Therapy for Huntington12.1. Intra-striatal rAAV5-miHTT: UniQure Biopharma12.1.1. Product Description12.1.2. Research and Development12.1.3. Product Developmental Activities13. Company Profiles For more information about this report visit