AAV

4DMT and Arbor Biotechnologies Establish Partnership to Co-Develop and Co-Commercialize Next-Generation Genetic Medicines for CNS Diseases

Retrieved on: 
Wednesday, January 3, 2024
Biology, Entrepreneurship, Partnership, ALS, Gene editing, Cardiology, A101, Drug development, R100, Protein engineering, FDA, CRISPR, Cystic fibrosis, Disease, Tissue, Brain, PAMS, Vivisection, AAV, Artificial intelligence, DSM-IV codes, Ophthalmology, CNS, Engineering, Machine learning, Genome, Central nervous system, Patient, Therapy, Vaccine

Arbor also has expertise in CNS biology, CNS drug development and CNS therapeutic development.

Key Points: 
  • Arbor also has expertise in CNS biology, CNS drug development and CNS therapeutic development.
  • 4DMT has utilized its platform to invent customized AAV vectors for CNS tissues, and these vectors will be deployed in the partnership.
  • “CNS disorders include some of the most devastating diseases, many of which have a genetic origin.
  • We look forward to combining our complementary technologies and capabilities to power a new generation of potential genetic medicines.

Canada Growth Fund Announces Strategic Investment in Entropy Inc. and Carbon Credit Offtake Commitment

Retrieved on: 
Thursday, December 21, 2023
Partnership, Public, Risk, Natural Resources, Sale, CGF, CCO, CCS, News, Investment, NEWS, TIER, AAV, Engineering, PSP, Growth, TSX, The Company, Partner (business rank), Renewable energy

CGF has agreed to a $200 million investment in Entropy coupled with a fixed-price carbon credit purchase agreement ("Carbon Credit Offtake Commitment" or "CCO") of up to one million tonnes per annum ("tpa").

Key Points: 
  • CGF has agreed to a $200 million investment in Entropy coupled with a fixed-price carbon credit purchase agreement ("Carbon Credit Offtake Commitment" or "CCO") of up to one million tonnes per annum ("tpa").
  • This strategic growth partnership represents an important new investment in Canadian carbon markets.
  • This financeable structure helps to de-risk and accelerate private CCS investment by establishing carbon price certainty for Canadian projects.
  • We welcome the additional capital and revenue certainty that the Canada Growth Fund is providing to enable Entropy's success."

Passage Bio Announces Promising Initial Data From Phase 1/2 Clinical Trial of PBFT02 in FTD-GRN and Updated Strategic Priorities

Retrieved on: 
Wednesday, December 20, 2023
CSF, Immunosuppression, Partnership, ALS, GM1, Aes, Krabbe disease, FTD, Loss, ICM, PASG, Clinical trial, Plasma, Inflammation, Methylprednisolone, AAV, Toxicity, GRN, Patient, Frontotemporal dementia, Safety, Cell biology, Hepatotoxicity, MLD, DRG, Pharmaceutical industry

Additionally, the company shared updated strategic priorities aimed at further optimizing its portfolio for the treatment of neurodegenerative conditions.

Key Points: 
  • Additionally, the company shared updated strategic priorities aimed at further optimizing its portfolio for the treatment of neurodegenerative conditions.
  • "We are proud to announce initial clinical data from our upliFT-D clinical trial, which showcases the ability of PBFT02 to elevate CSF progranulin to supraphysiologic levels at the lowest tested dose, Dose 1, up to six months post-treatment.
  • The upliFT-D clinical trial evaluates PBFT02 as a single dose delivered via intra-cisterna magna (ICM) injection.
  • These strategic priorities and clinical milestones underscore Passage Bio's dedication to advancing cutting-edge, one-time genetic medicines and protecting patients and families against loss in neurodegenerative conditions.

Business Update

Retrieved on: 
Thursday, December 7, 2023
Workforce, Conference, IND, Sale, Oxford Biomedica, Acquisition, 5L, European, CDMO, 7L, Investigational New Drug, Lentivirus, COVID-19, AAV, Annual, IMPD, Growth, OXB, Vaccine

Oxford Biomedica has made significant progress in its streamlining of operations to become a pure-play quality and innovation-led global cell and gene therapy CDMO.

Key Points: 
  • Oxford Biomedica has made significant progress in its streamlining of operations to become a pure-play quality and innovation-led global cell and gene therapy CDMO.
  • This organisational restructuring is tailored to boost efficiency and client-centricity, aligning roles and operations with the specific requirements of a pure-play CDMO.
  • The Company continues to demonstrate robust business development activity, a testament to its strong position in the viral vector space.
  • The Company also remains on track to deliver broadly breakeven Operating EBITDA in 2024, bolstered by positive momentum in business development activities and new orders recently secured across lentivirus, adenoviral vectors and AAV, with both new and existing clients.

4DMT to Present Interim 24 Week Data from Randomized Phase 2 PRISM Clinical Trial of 4D-150 in High Treatment Need Wet AMD Patients at Angiogenesis, Exudation, and Degeneration 2024 Conference

Retrieved on: 
Monday, December 4, 2023
Conference, AMD, Clinical trial, Medicine, University, M.A, AAV, University of Nevada, Reno, PRISM, Patient, Angiogenesis, Clinical research, Science

Initial interim landmark data analysis (N=50 at 24 Weeks) to be presented by Arshad M. Khanani, M.D., M.A., FASRS, Principal Investigator of the PRISM clinical trial, on Saturday, February 3, 2024 at 4:20 p.m.

Key Points: 
  • Initial interim landmark data analysis (N=50 at 24 Weeks) to be presented by Arshad M. Khanani, M.D., M.A., FASRS, Principal Investigator of the PRISM clinical trial, on Saturday, February 3, 2024 at 4:20 p.m.
  • ET
    EMERYVILLE, Calif., Dec. 04, 2023 (GLOBE NEWSWIRE) -- 4D Molecular Therapeutics (Nasdaq: FDMT, 4DMT or the Company), a clinical-stage biotherapeutics company with three novel, highly targeted next generation AAV vectors currently in the clinic, today announced that the initial interim landmark data analysis (24 weeks) from the randomized Dose Expansion stage (N=50) of the Phase 2 PRISM clinical trial in high anti-VEGF (vascular endothelial growth factor) treatment need (6-12 anti-VEGF injections in prior 12 months) wet AMD patients will be presented at the Angiogenesis, Exudation, and Degeneration 2024 Conference being held virtually on February 3, 2024.
  • The Company will also host a webcast to discuss the data in detail and include a 4D-150 program update with details to be announced at a future date.
  • Arshad M. Khanani, M.D., M.A., FASRS, Director of Clinical Research at Sierra Eye Associates, Clinical Associate Professor at University of Nevada, Reno School of Medicine
    The presentation from the Angiogenesis, Exudation, and Degeneration 2024 Conference will also be available on the 4D Molecular Therapeutics website under Scientific Presentations:

Taysha Gene Therapies Announces Expanded Eligibility in REVEAL Phase 1/2 Adult Trial to Include Adolescent Rett Syndrome Patients

Retrieved on: 
Wednesday, November 29, 2023
MTD, Adolescence, Index, IND, Rett syndrome, FDA, CMC, Ageing, Investigational New Drug, AAV, CNS, Regulatory affairs, Health Canada, Central nervous system, Patient, Safety, Rett, MECP2, Senior, Pharmaceutical industry

DALLAS, Nov. 29, 2023 (GLOBE NEWSWIRE) --  Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS), today announced that Health Canada has authorized the protocol amendment to the ongoing REVEAL Phase 1/2 adult trial evaluating TSHA-102 that expands eligibility to include patients aged 12 and older with Rett syndrome.

Key Points: 
  • DALLAS, Nov. 29, 2023 (GLOBE NEWSWIRE) --  Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS), today announced that Health Canada has authorized the protocol amendment to the ongoing REVEAL Phase 1/2 adult trial evaluating TSHA-102 that expands eligibility to include patients aged 12 and older with Rett syndrome.
  • “Following review of the initial clinical data from the first two adult patients treated with TSHA-102 and Chemistry, Manufacturing, and Controls (CMC) data, Health Canada has authorized our protocol amendment to include adolescent patients aged 12 years and older in the ongoing REVEAL Phase 1/2 adult trial,” said Sukumar Nagendran, M.D., President, and Head of R&D of Taysha.
  • “Amending our protocol broadens the patient population who can potentially benefit from TSHA-102.
  • The maximum tolerated dose (MTD) or maximum administered dose (MAD) established will then be administered during dose expansion.

Syncona to Acquire Freeline Therapeutics

Retrieved on: 
Wednesday, November 22, 2023
Slate, ADS, Committee, Acquisition, Gaucher's disease, Court, Proxy, SVB Securities, P.C, AAV, Part, Mass meeting, Scheme, Negotiation, Patient, Note, LON

LONDON, Nov. 22, 2023 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) today announced that it has entered into a definitive agreement with a newly established portfolio company of Syncona Ltd (LON: SYNC), a leading life science investor focused on creating, building and scaling global leaders in life science, to acquire Freeline in an all-cash transaction. Under the agreement, the newly established portfolio company will acquire all shares of Freeline not currently owned by Syncona for $6.50 per American Depositary Share (ADS). This price values Freeline’s entire issued share capital at approximately $28.3 million and represents a 51% premium over the closing price prior to the announcement of Syncona’s initial proposal on October 18. The acquisition is subject to the approval of Freeline’s minority shareholders and is expected to close in the first quarter of 2024.

Key Points: 
  • Under the agreement, the newly established portfolio company will acquire all shares of Freeline not currently owned by Syncona for $6.50 per American Depositary Share (ADS).
  • We are confident this negotiated transaction for Syncona to acquire Freeline and support it as a private entity is in the best interest of the company, as well as shareholders, employees and ultimately patients,” said Julia P. Gregory, an independent director and Chair of the Special Committee.
  • is acting as US legal counsel to Syncona; and Simmons & Simmons LLP is acting as UK legal counsel to Syncona.
  • Freeline entered into a secured convertible loan note in the aggregate amount of up to $15 million with Syncona (the “Note”).

JCR Pharmaceuticals and Takeda Pharmaceuticals Announce Discontinuation of Gene Therapy Collaboration

Retrieved on: 
Thursday, December 21, 2023
Health, Other Science, General Health, Research, Science, Pharmaceutical, Biotechnology, Research and development, Partnership, JCR, AAV, Brain, Glycogen storage disease, Medical device

JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) announced today that Takeda Pharmaceutical Co., Ltd. (“Takeda”) decided to discontinue their collaboration with JCR to develop gene therapies using adeno-associated viruses (AAV) combined with the JCR J-Brain Cargo® Technology.

Key Points: 
  • JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) announced today that Takeda Pharmaceutical Co., Ltd. (“Takeda”) decided to discontinue their collaboration with JCR to develop gene therapies using adeno-associated viruses (AAV) combined with the JCR J-Brain Cargo® Technology.
  • Under the agreement signed on March 28, 2022, JCR and Takeda collaborated on the research and development of AAV-mediated gene therapies that apply JCR’s J-Brain Cargo® technology.
  • The discontinuation is due to the change in Takeda's previously announced Research & Development (R&D) strategy to discontinue R&D on AAV-mediated gene therapy approaches.
  • “Through our collaboration, we secured proof-of-concept for a gene therapy using our proprietary J-Brain Cargo® technology platform,” said Shin Ashida, Chairman and President of JCR.

Genevoyager Unveils Gene Therapy CDMO Facility in Milestone Move

Retrieved on: 
Wednesday, December 20, 2023
FDA, EMA, CDMO, CGMP, Lentivirus, NMPA, AAV, A-DNA, Patient, Potyvirus, Medical device

PHILADELPHIA, Dec. 20, 2023 /PRNewswire/ -- Genevoyager (Wuhan) Co., Ltd. (Genevoyager), a leading provider of one-stop CRO/CDMO services for gene therapy products, proudly announces the official opening of its Contract Development and Manufacturing Organization (CDMO) facility.

Key Points: 
  • PHILADELPHIA, Dec. 20, 2023 /PRNewswire/ -- Genevoyager (Wuhan) Co., Ltd. (Genevoyager), a leading provider of one-stop CRO/CDMO services for gene therapy products, proudly announces the official opening of its Contract Development and Manufacturing Organization (CDMO) facility.
  • This significant milestone marks a major leap forward in the company's commitment to advancing healthcare through innovative gene therapy solutions.
  • Spanning 68,000 square feet, the facility is purpose-built to facilitate the seamless development, manufacturing, and scaling of AAV gene therapy products.
  • With the operation of the facility, Genevoyager will provide development and manufacturing services for AAV, lentivirus, oncolytic viruses, and recombinant proteins.

CANADA GROWTH FUND ANNOUNCES STRATEGIC INVESTMENT IN ENTROPY INC AND CARBON CREDIT OFFTAKE COMMITMENT

Retrieved on: 
Wednesday, December 20, 2023
AB, Partnership, Public, Risk, Natural Resources, Sale, CGF, CCO, CCS, News, Investment, TIER, AAV, Engineering, PSP, Growth, TSX, The Company, Partner (business rank), Renewable energy

CGF has agreed to a $200 million investment in Entropy coupled with a fixed-price carbon credit purchase agreement ("Carbon Credit Offtake Commitment" or "CCO") of up to one million tonnes per annum ("tpa").

Key Points: 
  • CGF has agreed to a $200 million investment in Entropy coupled with a fixed-price carbon credit purchase agreement ("Carbon Credit Offtake Commitment" or "CCO") of up to one million tonnes per annum ("tpa").
  • This strategic growth partnership represents an important new investment in Canadian carbon markets.
  • This financeable structure helps to de-risk and accelerate private CCS investment by establishing carbon price certainty for Canadian projects.
  • We welcome the additional capital and revenue certainty that the Canada Growth Fund is providing to enable Entropy's success."