Spinal muscular atrophy

New Biomarker Data Add Further Evidence Supporting the Potential Benefit of SPINRAZA® (nusinersen) in Infants and Toddlers with Unmet Clinical Needs after Gene Therapy

Retrieved on: 
Wednesday, March 6, 2024

* The Phase 4 study evaluates clinical outcomes and safety following treatment with SPINRAZA over a 2-year period in infants and toddlers with spinal muscular atrophy (SMA) who have unmet clinical needs after treatment with Zolgensma® (onasemnogene abeparvovec).

Key Points: 
  • * The Phase 4 study evaluates clinical outcomes and safety following treatment with SPINRAZA over a 2-year period in infants and toddlers with spinal muscular atrophy (SMA) who have unmet clinical needs after treatment with Zolgensma® (onasemnogene abeparvovec).
  • The new data show that plasma neurofilament light chain (NfL) levels, an objective biomarker of axonal injury and neurodegeneration, were reduced in nearly all study participants treated with SPINRAZA.
  • These data will be presented at the 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference (March 3-6, 2024).
  • “Our evolving understanding of gene therapy indicates there may be an opportunity for better outcomes,” said Crystal Proud, M.D., Pediatric Neurologist at Children’s Hospital of the King’s Daughters.

Fortress Biotech and Cyprium Therapeutics Announce $4.1 Million Grant from NINDS to Further Development of AAV-ATP7A Gene Therapy for Menkes Disease

Retrieved on: 
Monday, March 4, 2024

MIAMI, March 04, 2024 (GLOBE NEWSWIRE) -- Cyprium Therapeutics, Inc. (“Cyprium”), a majority-owned subsidiary of Fortress Biotech, Inc. (Nasdaq: FBIO) (“Fortress”), today announced that the National Institute of Neurological Disorders and Stroke (“NINDS”) of the National Institutes of Health (“NIH”) has awarded a three-year grant totaling approximately $4.1 million to the Research Institute at Nationwide Children’s Hospital and Principal Investigator, Stephen G. Kaler, M.D., M.P.H., to fund completion of preclinical studies, manufacturing and preparation of an Investigational New Drug Application for a first-in-human clinical trial to advance adeno-associated virus (“AAV”)-ATP7A gene therapy, also known as AAV-ATP7A, for the treatment of Menkes disease.

Key Points: 
  • Often lethal if untreated, Menkes disease is an X-linked recessive disorder of copper metabolism caused by mutations in ATP7A, an evolutionarily conserved copper-transporting ATPase.
  • “By combining CUTX-101 with working copies of ATP7A delivered by AAV, we hope to enhance clinical outcomes in Menkes disease, a fatal rare pediatric disease.
  • Preclinical studies have demonstrated a synergistic effect of AAV-ATP7A and CUTX-101 in a reliable mouse model of Menkes disease.
  • In early studies, cerebrospinal fluid (“CSF”)-directed AAV gene therapy rescued 22-53% of mice with a mutation in the human Menkes disease homolog (mottled-brindled) when combined with CSF or subcutaneous copper.

Voyager Therapeutics Reports Fourth Quarter and Full Year 2023 Financial and Operating Results

Retrieved on: 
Wednesday, February 28, 2024

LEXINGTON, Mass., Feb. 28, 2024 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to advancing neurogenetic medicines, today reported fourth quarter and full year 2023 financial and operating results.

Key Points: 
  • ET today -
    LEXINGTON, Mass., Feb. 28, 2024 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to advancing neurogenetic medicines, today reported fourth quarter and full year 2023 financial and operating results.
  • Novartis agreed to pay Voyager $80 million of consideration up front and $20 million for the purchase of newly issued equity in Voyager.
  • Collaboration Revenues: Voyager had collaboration revenue of $90.1 million for the fourth quarter of 2023, compared to $(1.6) million for the same period in 2022.
  • ET to discuss the fourth quarter and full year 2023 financial and operating results.

Scholar Rock Announces the Addition of Katie Peng to Its Board of Directors and Promotes Mo Qatanani, Ph.D. to Chief Scientific Officer

Retrieved on: 
Thursday, February 15, 2024

“We are pleased to welcome Katie to Scholar Rock’s Board of Directors at a pivotal time when the company is preparing for the potential launch of apitegromab next year,” said David Hallal, Chairman of the Board of Scholar Rock.

Key Points: 
  • “We are pleased to welcome Katie to Scholar Rock’s Board of Directors at a pivotal time when the company is preparing for the potential launch of apitegromab next year,” said David Hallal, Chairman of the Board of Scholar Rock.
  • Katie joining our Board reflects the continued evolution of Scholar Rock, and as part of this process, Amir Nashat will be stepping down from the Board at this year’s annual shareholder meeting.
  • Prior to joining Denali, Ms. Peng managed a neurology, ophthalmology, immunology, and rare disease portfolio representing approximately $14 billion in revenue at Genentech.
  • “I am thrilled to join Scholar Rock’s Board of Directors as the company prepares for its first potential commercial launch of apitegromab.

NMD Pharma Appoints Morten Bull as SVP, General Counsel and Head of People & Business Services

Retrieved on: 
Monday, January 22, 2024

NMD Pharma Appoints Morten Bull as SVP, General Counsel and Head of People & Business Services

Key Points: 
  • NMD Pharma Appoints Morten Bull as SVP, General Counsel and Head of People & Business Services
    Aarhus, Denmark, 22 January 2024 – NMD Pharma A/S, a clinical-stage biotech dedicated to developing novel and improved treatments for patients living with neuromuscular diseases, announces the appointment of Morten Bull as Senior Vice President, General Counsel and Head of People & Business Services with effect from 1 January 2024.
  • He has been working with NMD Pharma since June 2023 as General Counsel on a consultancy basis prior to this appointment.
  • Before joining NMD Pharma, Morten was Executive Director and Global Head of IP and R&D Legal of LEO Pharma, where he was part of the Legal and R&D leadership teams and business partner to the M&A group.
  • Morten Bull, newly appointed SVP, General Counsel and Head of People & Business Services at NMD Pharma, said: “I have been working closely with NMD Pharma since June 2023 and am very pleased to now be joining the Company on a fulltime basis as SVP, General Counsel and Head of People & Business Services.

Scholar Rock Provides Corporate Update and Highlights Priorities for 2024

Retrieved on: 
Thursday, January 4, 2024

Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on advancing innovative treatments for spinal muscular atrophy (SMA), cardiometabolic disorders, and other serious diseases where protein growth factors play a fundamental role, today provided recent corporate updates and highlighted upcoming priorities for 2024.

Key Points: 
  • Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on advancing innovative treatments for spinal muscular atrophy (SMA), cardiometabolic disorders, and other serious diseases where protein growth factors play a fundamental role, today provided recent corporate updates and highlighted upcoming priorities for 2024.
  • Planning to announce Phase 3 SAPPHIRE clinical trial topline data in 4Q 2024.
  • Scholar Rock management will highlight these updates in a corporate presentation at the 42nd Annual J.P. Morgan Healthcare Conference on Tuesday, January 9, 2024, at 1:30 p.m. PT (4:30 p.m.
  • A live webcast of the presentation may be accessed by visiting the Investors & Media section of the Scholar Rock website at http://investors.scholarrock.com .

Human medicines European public assessment report (EPAR): Zolgensma, onasemnogene abeparvovec, Date of authorisation: 18/05/2020, Revision: 14, Status: Authorised

Retrieved on: 
Tuesday, January 2, 2024

Human medicines European public assessment report (EPAR): Zolgensma, onasemnogene abeparvovec, Date of authorisation: 18/05/2020, Revision: 14, Status: Authorised

Key Points: 


Human medicines European public assessment report (EPAR): Zolgensma, onasemnogene abeparvovec, Date of authorisation: 18/05/2020, Revision: 14, Status: Authorised

Voyager Therapeutics Enters Capsid License Agreement and Strategic Collaboration with Novartis to Advance Novel Gene Therapies

Retrieved on: 
Tuesday, January 2, 2024

LEXINGTON, Mass., Jan. 02, 2024 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to advancing neurogenetic medicines, today announced a strategic collaboration and capsid license agreement with Novartis Pharma AG, a subsidiary of Novartis AG (NYSE: NVS) to advance potential gene therapies for Huntington’s disease (HD) and spinal muscular atrophy (SMA). Voyager will provide Novartis a target-exclusive license to access Voyager’s TRACER™ capsids and other intellectual property for the respective diseases, and Voyager and Novartis will collaborate to advance a preclinical gene therapy candidate for HD.

Key Points: 
  • LEXINGTON, Mass., Jan. 02, 2024 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to advancing neurogenetic medicines, today announced a strategic collaboration and capsid license agreement with Novartis Pharma AG, a subsidiary of Novartis AG (NYSE: NVS) to advance potential gene therapies for Huntington’s disease (HD) and spinal muscular atrophy (SMA).
  • Voyager will provide Novartis a target-exclusive license to access Voyager’s TRACER™ capsids and other intellectual property for the respective diseases, and Voyager and Novartis will collaborate to advance a preclinical gene therapy candidate for HD.
  • “We are thrilled to expand our existing relationship with Novartis, a global leader in the gene therapy field,” said Alfred W. Sandrock, Jr., M.D., Ph.D., Chief Executive Officer of Voyager.
  • Novartis will also receive worldwide rights to Voyager’s AAV gene therapy for HD, leveraging Voyager’s TRACER capsids and proprietary payloads.

NMD Pharma Raises €75 Million (~$80 million) in a Series B Financing

Retrieved on: 
Wednesday, November 15, 2023

The financing was led by current investor Jeito Capital and includes investments from other current NMD Pharma investors: Novo Holdings, Lundbeckfonden BioCapital, INKEF Capital, and the Roche Venture Fund.

Key Points: 
  • The financing was led by current investor Jeito Capital and includes investments from other current NMD Pharma investors: Novo Holdings, Lundbeckfonden BioCapital, INKEF Capital, and the Roche Venture Fund.
  • NMD Pharma is developing first-in-class small molecule inhibitors of the skeletal muscle specific chloride ion channel (ClC-1) to enhance neuromuscular transmission with the potential to restore muscle function in a range of rare neuromuscular diseases.
  • Proceeds from the financing will be used to complete three Phase 2 studies with NMD670, the Company’s lead ClC-1 inhibitor.
  • Furthermore, NMD Pharma will continue to expand its pipeline of ClC-1 inhibitor molecules and pursue undisclosed targets for the treatment of other neuromuscular diseases.

OXFORD PROPERTIES BREAKS GROUND ON 165,000 SQ FT EXPANSION OF IONIS PHARMACEUTICALS CARLSBAD CAMPUS

Retrieved on: 
Tuesday, December 5, 2023

Located in the San Diego life sciences submarket of Carlsbad, Ionis' existing campus features approximately 250,000 square feet of lab and office space.

Key Points: 
  • Located in the San Diego life sciences submarket of Carlsbad, Ionis' existing campus features approximately 250,000 square feet of lab and office space.
  • The expansion builds on Oxford and Ionis' established partnership, with Oxford completing a purchase and long-term lease back of Ionis' existing campus in October 2022.
  • Oxford entered the San Diego life sciences market in February 2022 via a US$464 million acquisition of a 13-building portfolio.
  • The campus expansion supports Ionis' strategic goals of prioritizing its wholly owned pipeline and advancing its drug discovery technology.