Duchenne

Santhera Announces Preliminary Unaudited 2023 Annual Results and Provides Corporate Update Ahead of Full Report Publication in May

Retrieved on: 
Thursday, April 25, 2024
FDA, Annual, Pharmacy, NMPA, Private, Partnership, CDE, PDUFA, CEPS, European Medicines Agency, News, Annual report, Intangibles, DMD, U.S. FDA, Catalyst Pharmaceuticals, Tax, CHF, MHRA, Duchenne muscular dystrophy, Quality of life, Leber's hereditary optic neuropathy, Vamorolone, BST, Public, EMA, Marketing, File, Sale, Medication, Treasury, NDA, Health care, Duchenne, Bond, Patient, Bank statement, AGM, Growth, Cryptocurrency

As permitted by SIX Exchange Regulation, the Company will publish the full 2023 Annual Report in May.

Key Points: 
  • As permitted by SIX Exchange Regulation, the Company will publish the full 2023 Annual Report in May.
  • Financially, Santhera reported a 2023 revenue of CHF 103.4 million and net income of CHF 54.8 million, driven by the Catalyst licensing deal.
  • After closing of the transaction in July 2023, Santhera received an upfront payment of USD 90 million (USD 75 million in cash and USD 15 million equity investment).
  • SIX Exchange Regulation has permitted Santhera to publish its 2023 Annual Report by May 31, 2024, at the latest.

U.S. FDA Approves Pfizer’s BEQVEZ™ (fidanacogene elaparvovec-dzkt), a One-Time Gene Therapy for Adults with Hemophilia B

Retrieved on: 
Friday, April 26, 2024
Biotechnology, FDA, Health, Pharmaceutical, Clinical Trials, World government, FIX, Risk, Food, Love, Duchenne, Coalition, Biologics license application, Bleeding, Disease, Patient, PFE, EMA, Medicine, Inc., Standard of care, Duchenne muscular dystrophy, Haemophilia B, European Medicines Agency, Pharmaceutical industry

“For people living with hemophilia, disease management can interfere with many aspects of their lives.

Key Points: 
  • “For people living with hemophilia, disease management can interfere with many aspects of their lives.
  • A one-time infusion of BEQVEZ may allow eligible patients more time for the things they love,” said Kim Phelan, Chief Operating Officer, The Coalition for Hemophilia B.
  • “We are excited to have BEQVEZ as a promising treatment option for eligible people living with hemophilia B.
  • A Biologics License Application and European Marketing Authorization Application for marstacimab are currently under review with the FDA and EMA, respectively.

First Clinical Trial Results of Gene Therapy (GNT0004) for Duchenne Muscular Dystrophy Presented at International Myology 2024 Congress

Retrieved on: 
Tuesday, April 23, 2024
Science, Biotechnology, Research, Pharmaceutical, General Health, Health, Genetics, Clinical Trials, Safety, Myology, Dystrophin-associated protein complex, CPK, NSAA, Immunohistochemistry, Duchenne, Duchenne muscular dystrophy, Disease, Patient, Ageing, Pharmaceutical industry

Today, Professor Francesco Muntoni, principal investigator of the international multicenter gene therapy trial for Duchenne Muscular Dystrophy with GNT0004 product, sponsored by Genethon, presented the innovative trial design and its initial results at the Myology 2024 international scientific congress, currently taking place in Paris.

Key Points: 
  • Today, Professor Francesco Muntoni, principal investigator of the international multicenter gene therapy trial for Duchenne Muscular Dystrophy with GNT0004 product, sponsored by Genethon, presented the innovative trial design and its initial results at the Myology 2024 international scientific congress, currently taking place in Paris.
  • The trial combines phases I/II/III with a dose escalation phase, followed by a pivotal phase at the selected dose.
  • The trial has been approved by French and UK health authorities and includes ambulant boys aged 6 to 10 suffering from Duchenne Muscular Dystrophy.
  • Note that the dose chosen is lower than those used in other gene therapy trials for Duchenne muscular dystrophy.

Edgewise Therapeutics Announces Positive Two-Year Topline Results from the ARCH Open Label Trial of Sevasemten (EDG-5506) in Adults with Becker Muscular Dystrophy (Becker)

Retrieved on: 
Monday, April 15, 2024
Biotechnology, Pharmaceutical, Health, Clinical Trials, TNNI2, Neuromuscular disease, University, Safety, Pharmacokinetics, MDA, Muscle weakness, NSAA, Duchenne, Patient, Muscular dystrophy, University of Florida, Multimedia, CK, Duchenne muscular dystrophy, Myopathy, Biomarker, Therapy, ARCH, Pharmaceutical industry

Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced positive two-year topline results from the ARCH trial.

Key Points: 
  • Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced positive two-year topline results from the ARCH trial.
  • ARCH is an open label, single-center study assessing safety, tolerability, impact on muscle damage biomarkers, pharmacokinetics (PK) and functional measures with sevasemten (EDG-5506) in adults with Becker.
  • Sevasemten is an orally administered small molecule designed to prevent contraction-induced muscle damage in dystrophinopathies including Becker and Duchenne muscular dystrophy (Duchenne).
  • ET to discuss the ARCH two-year data, and will be joined by Dr. Byrne, who will share his perspective of sevasemten and Becker.

MUSCLE CULTIVATION STARTUP PROFUSE-TECHNOLOGY WELCOMES RENOWNED TECHNION PROFESSOR TO ITS SCIENTIFIC ADVISORY BOARD

Retrieved on: 
Monday, April 22, 2024
Diabetes, Biomedical engineering, Weight loss, Research, Duchenne, Weizmann Institute of Science, Obesity, Biomedicine, Therapy, Cachexia, Patient, Tissue engineering, Regenerative medicine, Sarcopenia, Duchenne muscular dystrophy, Faculty, Ageing, Biotechnology, Pharmaceutical industry

YOKNEAM, Israel, April 22, 2024 /PRNewswire/ -- ProFuse Technology expands its scope as a muscle cultivation startup aiming to enhance patient outcomes by improving treatments for muscle associated diseases and muscle regeneration. The company has appointed Professor Shulamit Levenberg of the Technion Faculty of Biomedical Engineering to its Scientific Advisory Board, to support its effort towards life science applications.

Key Points: 
  • YOKNEAM, Israel, April 22, 2024 /PRNewswire/ -- ProFuse Technology expands its scope as a muscle cultivation startup aiming to enhance patient outcomes by improving treatments for muscle associated diseases and muscle regeneration.
  • The company has appointed Professor Shulamit Levenberg of the Technion Faculty of Biomedical Engineering to its Scientific Advisory Board, to support its effort towards life science applications.
  • She has joined ProFuse Technology's Scientific Advisory Board to bolster the company's effort to develop materials and methodologies for muscle regeneration and maintenance.
  • We are thrilled to welcome Professor Levenberg's to our Scientific Advisory Board, in order to bolster our pioneering innovative approaches towards muscle regeneration and maintenance."

Solid Biosciences Receives Rare Pediatric Disease Designation from the FDA for Duchenne Muscular Dystrophy Gene Therapy Candidate SGT-003

Retrieved on: 
Monday, April 1, 2024
Patient, DNA, Hope, Tropism, Duchenne muscular dystrophy, Duchenne, NOS1, Safety, FDA, DMD, Food, Pharmaceutical industry

CHARLESTOWN, Mass., April 01, 2024 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation for SGT-003, the company’s next-generation Duchenne muscular dystrophy (Duchenne) gene therapy candidate.

Key Points: 
  • “Solid’s receipt of Rare Pediatric Disease Designation for SGT-003 highlights the continuing need for transformational treatments for this devastating disease,” said Bo Cumbo, President and Chief Executive Officer at Solid Biosciences.
  • “The key components of SGT-003 were rationally designed to improve on first generation gene therapies to provide skeletal muscle tropism, enhanced durability, and improved clinical outcomes.
  • nNOS is believed to play a crucial role in both muscular function and endurance,” said Dr. Gabriel Brooks, M.D., Chief Medical Officer at Solid Biosciences.
  • “We look forward to rapidly bringing SGT-003 to the clinic and hope to all Duchenne patients in need.”

Burn Boot Camp and Muscular Dystrophy Association Team Up for 8th Annual 'Be Their Muscle' Philanthropic Event

Retrieved on: 
Monday, March 25, 2024
Research, Employment, Entrepreneurship, Skeletal muscle, MDA, ALS, Partnership, Air travel, Education, Duke University Hospital, Social media, Muscular dystrophy, Muscular Dystrophy Association, Senior, Empowerment, Duchenne muscular dystrophy, Duchenne, Clinical trial, FDA, Summer camp, Pharmaceutical industry

Charlotte, North Carolina, March 25, 2024 (GLOBE NEWSWIRE) -- Burn Boot Camp, a leading boutique fitness franchise, has teamed up with the Muscular Dystrophy Association (MDA) for its eighth annual national 'Be Their Muscle' philanthropic event.

Key Points: 
  • Charlotte, North Carolina, March 25, 2024 (GLOBE NEWSWIRE) -- Burn Boot Camp, a leading boutique fitness franchise, has teamed up with the Muscular Dystrophy Association (MDA) for its eighth annual national 'Be Their Muscle' philanthropic event.
  • Muscular Dystrophy Association is one of Burn Boot Camp's longest-standing national philanthropic partners," explained Morgan Kline, CEO and Co-Founder of Burn Boot Camp.
  • "Here at the Muscular Dystrophy Association, we stand hand in hand with Burn Boot Camp in our shared commitment to empower communities nationwide.
  • MDA and Burn Boot Camp will be posting throughout the campaign on social media using @MDAorg and @burnbootcamp with #BeTheirMuscle.

PepGen Receives U.S. FDA Orphan Drug and Rare Pediatric Disease Designations for PGN-EDO51 for the Treatment of Duchenne Muscular Dystrophy

Retrieved on: 
Wednesday, March 13, 2024
Disease, Patient, Ageing, Duchenne muscular dystrophy, Duchenne, FDA, DMD, Food, Pharmaceutical industry

“Receiving these FDA designations for PGN-EDO51 emphasizes the critical demand for novel and enhanced therapeutic options for people living with DMD,” said James McArthur, Ph.D., President and CEO of PepGen.

Key Points: 
  • “Receiving these FDA designations for PGN-EDO51 emphasizes the critical demand for novel and enhanced therapeutic options for people living with DMD,” said James McArthur, Ph.D., President and CEO of PepGen.
  • The FDA defines a "rare pediatric disease" as a serious or life-threatening disease in which the serious or life-threatening manifestations primarily affect individuals aged from birth to 18 years.
  • Under the Rare Pediatric Disease program, the FDA may award priority review vouchers to sponsors who have been granted rare pediatic disease designation for its drug.
  • For more information on these designations, please visit the FDA website’s Orphan Drug and Rare Pediatric Disease webpages.

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: 
Thursday, March 7, 2024
Liver, Duchenne, Toxicity, Inflammation, DMD, Facioscapulohumeral muscular dystrophy, DUX4, Solid, FSHD, Capsid, MicroRNA, Oxidative stress, B cell, IND, RNA interference, AAV, Atrophy, Vaccine

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

Retrieved on: 
Monday, March 4, 2024
Duchenne, Messenger, DMD, Duchenne muscular dystrophy, Entrepreneurship, Conference, RNA, Therapy, Muscle weakness, MDA, Atrophy, Episcopal conference, Vaccine

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.”