Duchenne

Solid Biosciences Announces Licensing Agreement with Armatus Bio for the Use of AAV-SLB101, a Proprietary, Muscle-Targeted Capsid, in the Development of an RNAi Therapy to treat FSHD

Retrieved on: 
Jeudi, mars 7, 2024

The AAV-SLB101 capsid has been shown in preclinical studies to have enhanced biodistribution and improved expression in muscle cells.

Key Points: 
  • The AAV-SLB101 capsid has been shown in preclinical studies to have enhanced biodistribution and improved expression in muscle cells.
  • Under the terms of the agreement, Solid granted Armatus a non-exclusive worldwide license to utilize AAV-SLB101 for treatment of FSHD and will provide Armatus AAV-SLB101 plasmid material, preclinical data characterizing AAV-SLB101, and manufacturing and regulatory know-how to enable development.
  • Armatus will be responsible for the development and commercialization of licensed products incorporating AAV-SLB101.
  • With this license in place, we are eager to advance our optimized ARM-201 construct as the lead candidate toward clinical evaluation.”

Elixirgen Therapeutics Presents Preclinical Data with Bobcat mRNATM Technology in Duchenne Muscular Dystrophy at the 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference

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Lundi, mars 4, 2024

BALTIMORE, March 04, 2024 (GLOBE NEWSWIRE) -- Elixirgen Therapeutics, Inc., a clinical-stage biotechnology company focused on the discovery, development and commercialization of therapies using its mRNA platforms, today presented preclinical data on its proprietary Bobcat mRNATM technology in a poster presentation at the 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference taking place March 3-6, 2024, in Orlando, Florida. Bobcat mRNATM is designed to deliver large protein payloads (over 13 kb), and results show that a Bobcat mRNATM encoding a full-length dystrophin protein can restore muscular function in a mouse model for Duchenne muscular dystrophy (DMD).

Key Points: 
  • Bobcat mRNATM is designed to deliver large protein payloads (over 13 kb), and results show that a Bobcat mRNATM encoding a full-length dystrophin protein can restore muscular function in a mouse model for Duchenne muscular dystrophy (DMD).
  • “We’re excited to be presenting these promising data on our novel Bobcat mRNATM technology.
  • “Our Bobcat mRNATM full-length dystrophin approach may be complementary to others currently approved or in development, or may even mitigate some of their issues.
  • We’re pleased to add the Bobcat mRNATM technology to our pipeline of RNA technologies and look forward to sharing updates in the months ahead.”

Genethon Pursues Different Strategies for Ensuring Patient Access to Gene Therapies for Rare Diseases

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Jeudi, février 29, 2024

Genethon , a non-profit research organization focused on developing gene therapies for rare diseases, today marks International Rare Disease Day by highlighting its efforts to bring gene therapies to patients suffering from rare diseases such as limb girdle muscular dystrophies, Crigler Najjar syndrome and Duchenne muscular dystrophy.

Key Points: 
  • Genethon , a non-profit research organization focused on developing gene therapies for rare diseases, today marks International Rare Disease Day by highlighting its efforts to bring gene therapies to patients suffering from rare diseases such as limb girdle muscular dystrophies, Crigler Najjar syndrome and Duchenne muscular dystrophy.
  • Globally more than 300 million people, most of them children, are living with 7,000 rare diseases.
  • Because patient populations are small, these diseases don’t readily fit into the biopharma industry’s business models.
  • “Despite challenges in generating interest with biopharma companies and investors, Genethon will never abandon patients suffering from rare and ultra-rare diseases,” said Dr. Revah.

Sarepta Therapeutics Announces Call for Applications for the 7th Annual Route 79, The Duchenne Scholarship Program

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Jeudi, février 29, 2024

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced the official opening of Route 79, The Duchenne Scholarship Program for the 2024-2025 academic year.

Key Points: 
  • Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced the official opening of Route 79, The Duchenne Scholarship Program for the 2024-2025 academic year.
  • “Sarepta is proud to announce the opening of Route 79, The Duchenne Scholarship Program for the 2024-2025 academic year.
  • Only applicants diagnosed with Duchenne or applicants who have a sibling diagnosed with Duchenne are eligible for the program.
  • Previous recipients of Route 79 scholarships may apply for the 2024 Scholarship Program, however, applicants may only receive a Route 79 scholarship up to four times.

NS Pharma, Inc. Shares New VILTEPSO® (Viltolarsen) Data at the MDA Clinical & Scientific Conference 2024

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Mercredi, mars 6, 2024

PARAMUS, N.J., March 6, 2024 /PRNewswire/ -- NS Pharma, Inc. (NS Pharma) is excited to announce participation in the 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference in Orlando, Florida, March 3 – 6. The company presented a poster entitled "Pulmonary and motor function in ambulatory and non-ambulatory participants with Duchenne muscular dystrophy (Duchenne) treated with viltolarsen (VILTEPSO®)" which covers data from the Galactic53 trial demonstrating that the majority of participants receiving viltolarsen experienced meaningful benefit in pulmonary function, including percent predicted forced vital capacity (FVC%p).

Key Points: 
  • PARAMUS, N.J., March 6, 2024 /PRNewswire/ -- NS Pharma, Inc. (NS Pharma) is excited to announce participation in the 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference in Orlando, Florida, March 3 – 6.
  • "Galactic53 is the first trial with VILTEPSO to evaluate pulmonary function in participants with Duchenne," explains NS Pharma Vice President Medical Affairs & Pharmacovigilance Leslie Magnus, MD, who also co-authored the poster.
  • "Our team is encouraged by these results and will continue our research into treatments for rare disease."
  • Additional data from this study will also be presented at the American Academy of Neurology (AAN) 2024 Annual Meeting, April 13 – 18 in Denver, Colorado and online.

Satellos to Commence Trading on the Toronto Stock Exchange

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Mercredi, février 14, 2024

Satellos Bioscience Inc. (“Satellos” or the “Company”) (TSX: MSCL) (OTCQB: MSCLF), a public biotech company developing new small molecule therapeutic approaches to improve the treatment of muscle diseases and disorders, announced today that its common shares will commence trading on the Toronto Stock Exchange (“TSX”) as of the opening of trading tomorrow under the Company’s current trading symbol, “MSCL”.

Key Points: 
  • Satellos Bioscience Inc. (“Satellos” or the “Company”) (TSX: MSCL) (OTCQB: MSCLF), a public biotech company developing new small molecule therapeutic approaches to improve the treatment of muscle diseases and disorders, announced today that its common shares will commence trading on the Toronto Stock Exchange (“TSX”) as of the opening of trading tomorrow under the Company’s current trading symbol, “MSCL”.
  • “We are excited to complete the uplist to the Toronto Stock Exchange, which will provide access to a broader set of institutional investors from around the world,” said Frank Gleeson, Co-founder and CEO of Satellos.
  • “This uplisting reflects the milestones we have achieved with our accomplished team at Satellos as we transition from a preclinical to a clinical-stage company.
  • We look forward to achieving a number of key milestones this year as we look to advance our lead small molecule drug candidate for Duchenne muscular dystrophy into clinical trials and explore the potential of our small molecule regenerative medicine approach in other degenerative muscle diseases.”
    Shareholders are not required to exchange their share certificates or direct registration system advices, or take any other action in connection with the listing on the TSX, as there will be no change in the trading symbol or CUSIP for the Common Shares.

Parent Project Muscular Dystrophy Hosts 2024 Duchenne Healthcare Professionals Summit in Sanibel, Florida

Retrieved on: 
Mercredi, janvier 17, 2024

WASHINGTON, Jan. 17, 2024 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD) , a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne) , will host its fifth in-person Duchenne Healthcare Professionals Summit in Sanibel, Florida, January 17-19.

Key Points: 
  • WASHINGTON, Jan. 17, 2024 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD) , a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne) , will host its fifth in-person Duchenne Healthcare Professionals Summit in Sanibel, Florida, January 17-19.
  • Since the Summit's inception in 2018, the event has brought together thousands of healthcare providers, researchers, leaders in federal advocacy, and industry partners for discussion, collaboration, and connection.
  • "Our hope is continued investment in these efforts will lead to improved care for every person and every family living with Duchenne and Becker."
  • Additionally,  learnings from the 2024 Summit will be shared at PPMD's 30th Annual Conference this summer in Orlando, Florida.

Solid Biosciences Granted FDA Orphan Drug Designation for Duchenne Muscular Dystrophy Gene Therapy Candidate SGT-003

Retrieved on: 
Mardi, janvier 16, 2024

CHARLESTOWN, Mass., Jan. 16, 2024 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced that it has been granted orphan drug designation (ODD) from the U.S. Food and Drug Administration (FDA) for SGT-003, the company’s next-generation Duchenne muscular dystrophy (Duchenne) gene therapy candidate.

Key Points: 
  • CHARLESTOWN, Mass., Jan. 16, 2024 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced that it has been granted orphan drug designation (ODD) from the U.S. Food and Drug Administration (FDA) for SGT-003, the company’s next-generation Duchenne muscular dystrophy (Duchenne) gene therapy candidate.
  • “Obtaining ODD status for SGT-003, along with Fast Track Designation granted last month, furthers our efforts to meet the ongoing challenge of treating this devastating disease as expeditiously as possible,” said Bo Cumbo, President and Chief Executive Officer at Solid Biosciences.
  • “SGT-003 therapy stands out among other Duchenne gene therapy candidates by leveraging a novel capsid and a muscle tropic vector delivering a microdystrophin that incorporates a neuronal Nitric Oxide Synthase (nNOS) binding domain.
  • “Obtaining ODD status is an exciting development that we believe will aid our efforts to bring advanced treatment options to those patients affected by Duchenne.”

Satellos Receives Conditional Approval to Graduate to the Toronto Stock Exchange

Retrieved on: 
Mardi, janvier 16, 2024

Satellos Bioscience Inc. (“Satellos” or the “Company”) (TSXV: MSCL) (OTCQB: MSCLF), a public biotech company developing new small molecule therapeutic approaches to improve the treatment of muscle diseases and disorders, is pleased to announce that it has received conditional approval from the Toronto Stock Exchange (“TSX”) to graduate from the TSX Venture Exchange (the “TSXV”) and to list its common shares (the “Common Shares”) on the TSX.

Key Points: 
  • Satellos Bioscience Inc. (“Satellos” or the “Company”) (TSXV: MSCL) (OTCQB: MSCLF), a public biotech company developing new small molecule therapeutic approaches to improve the treatment of muscle diseases and disorders, is pleased to announce that it has received conditional approval from the Toronto Stock Exchange (“TSX”) to graduate from the TSX Venture Exchange (the “TSXV”) and to list its common shares (the “Common Shares”) on the TSX.
  • We remain on target to initiate clinical trials mid-2024.”
    Final approval of the listing is subject to the Company meeting certain customary conditions required by the TSX.
  • The Company will issue a press release once the TSX confirms the date when trading of the Common Shares is expected to commence on the TSX.
  • Upon completion of the final TSX listing requirements, the Common Shares will be delisted from the TSXV.

Sarepta Therapeutics Reports Preliminary* Fourth Quarter and Full-Year 2023 Net Product Revenue

Retrieved on: 
Lundi, janvier 8, 2024

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today reported preliminary* fourth quarter and full-year 2023 net product revenue and cash on hand as of December 31, 2023, as part of its presentation today at the 42nd Annual J.P. Morgan Healthcare Conference in San Francisco, Calif.

Key Points: 
  • Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today reported preliminary* fourth quarter and full-year 2023 net product revenue and cash on hand as of December 31, 2023, as part of its presentation today at the 42nd Annual J.P. Morgan Healthcare Conference in San Francisco, Calif.
    Fourth quarter and full-year 2023 net product revenue for ELEVIDYS are expected to be approximately $131.3 million and $200.4 million, respectively.
  • Sarepta’s expected net product revenue does not include collaboration revenue.
  • Fourth quarter and full-year 2023 net product revenue for Sarepta’s RNA-based PMOs are expected to be approximately $234.3 million and $945.0 million, respectively.
  • Sarepta will report its final and complete fourth quarter and full-year 2023 financial results in late February 2024.