GENETHON | MedPath

Wave Life Sciences to Seek FDA Approval for Duchenne Muscular Dystrophy Drug Following Promising Phase 2 Results

2 months ago

• Wave Life Sciences will pursue FDA accelerated approval for WVE-N531, an exon 53 skipping therapy for Duchenne muscular dystrophy, following positive Phase 2 trial results showing increased dystrophin production. • The experimental treatment demonstrated substantial improvements in muscle health with a statistically significant improvement in "time to rise" functional tests compared to historical controls through 48 weeks of treatment. • If approved, WVE-N531 could offer a potential monthly dosing regimen and become a new therapeutic option for approximately 8-10% of Duchenne patients with specific genetic mutations amenable to exon 53 skipping.

AAV Gene Therapy Market Projected to Reach $107.2 Billion by 2035, Growing at 40% CAGR

3 months ago

• The adeno-associated virus (AAV) gene therapy market is expected to surge from $2.7 billion in 2024 to $107.2 billion by 2035, driven by advances in genetics, biotechnology, and personalized medicine. • Marketed AAV therapies include Luxturna for hereditary retinal dystrophy, Zolgensma for spinal muscular atrophy, and Glybera for lipoprotein lipase deficiency, with numerous promising candidates in late-stage development. • Improved diagnostic technologies, regulatory support, and significant investment from companies like Spark Therapeutics, Novartis, and BioMarin are accelerating the development of next-generation AAV therapies for previously untreatable genetic disorders.

Solid Biosciences' SGT-003 Gene Therapy Shows Promise in Duchenne Muscular Dystrophy Trial

4 months ago

• Solid Biosciences reported positive initial data from its Phase 1/2 INSPIRE DUCHENNE trial of SGT-003, a gene therapy candidate for Duchenne muscular dystrophy (DMD). • The trial showed an average microdystrophin expression of 110% in the first three patients, along with improvements in biomarkers indicating muscle health and resilience. • SGT-003 was well-tolerated in the first six participants, with adverse events typical of AAV gene therapy and no serious safety concerns reported. • Solid Biosciences plans to meet with the FDA to discuss a potential accelerated approval pathway for SGT-003 based on these encouraging early results.

Hansa Biopharma's Imlifidase Shows Promise in Guillain-Barré Syndrome and Anti-GBM Disease

4 months ago

• Hansa Biopharma announced positive Phase 2 results for imlifidase in Guillain-Barré Syndrome (GBS), showing rapid functional improvement in patients. • The company completed enrollment in the pivotal Phase 3 GOOD-IDES-02 trial of imlifidase for anti-glomerular basement membrane (anti-GBM) disease. • A Phase 2 trial of imlifidase as a pre-treatment to GNT-0003 in severe Crigler-Najjar syndrome has been initiated in collaboration with Genethon. • Hansa Biopharma anticipates submitting a Biologics License Application (BLA) to the FDA for imlifidase in kidney transplantation in late 2025.

Key Advances in Neuromuscular and Movement Disorder Treatments Highlighted in 2024

5 months ago

• AbbVie's Vyalev, a 24-hour subcutaneous levodopa infusion, gained FDA approval for managing motor fluctuations in advanced Parkinson's, offering a novel therapeutic approach. • Intellia's CRISPR therapy, NTLA-2001, demonstrated safe redosing in ATTR amyloidosis patients, achieving additive pharmacodynamic effects on the target protein. • A phase 3 study revealed that buntanetap is a safe and well-tolerated drug which improves motor, nonmotor, and cognitive symptoms of Parkinson's disease. • The FDA supported using αSyn-SAA biomarker in Parkinson's clinical trials, enhancing therapeutic development through improved diagnostic precision.

Elevidys Gene Therapy Shows Sustained Benefits in Duchenne Muscular Dystrophy Patients

5 months ago

• Sarepta Therapeutics' Elevidys demonstrates sustained benefits and disease stabilization in ambulatory Duchenne muscular dystrophy (DMD) patients, according to Phase 3 EMBARK trial results. • Crossover-treated patients showed a 2.34-point improvement on the North Star Ambulatory Assessment (NSAA) compared to matched external controls after 52 weeks of Elevidys treatment. • Patients treated with Elevidys in Part 1 of EMBARK maintained clinically meaningful improvements in NSAA, Time to Rise (TTR), and 10-meter walk/run (10MWR) at two years. • Muscle biopsies showed consistent micro-dystrophin expression, and MRI scans indicated minimal muscle pathology progression, reinforcing Elevidys's long-term efficacy and safety.

Imlifidase Shows Promise in Guillain-Barré Syndrome and Expands Gene Therapy Access

6 months ago

• Phase 2 data indicates imlifidase, combined with IVIg, significantly improves motor function and accelerates recovery in Guillain-Barré Syndrome (GBS) patients. • An indirect comparison showed GBS patients treated with imlifidase and IVIg returned to independent walking six weeks sooner than those treated with IVIg alone. • Hansa Biopharma and Genethon initiated a Phase 2 trial using imlifidase as a pretreatment to broaden access to gene therapy for Crigler-Najjar syndrome. • Hansa Biopharma completed enrollment in a Phase 3 trial of imlifidase for anti-GBM disease, with data expected in 2025.

Genethon's GNT0004 Gene Therapy Shows Promise in Duchenne Muscular Dystrophy Trial

6 months ago

• Genethon's GNT0004, a gene therapy for Duchenne muscular dystrophy (DMD), demonstrates positive initial results in a Phase 1/2/3 clinical trial, showing stable or improved motor function in treated patients. • The therapy delivers a gene encoding microdystrophin, a shortened but functional version of the dystrophin protein, leading to an average of 54% expression in muscle fibers eight weeks post-administration. • Creatine phosphokinase levels, a marker for muscle damage, decreased by an average of 74% twelve weeks post-therapy, remaining low for up to 18 months in evaluated patients. • Genethon plans to initiate a Phase 3 pivotal trial in Europe in Q2 2025, followed by the U.S., with potential for regulatory approval based on positive trial outcomes.

Gene Therapy Advances: From FDA Clearances to Clinical Trial Progress in Early 2025

6 months ago

• The FDA cleared United Therapeutics' UKidney for US trials, a gene-edited porcine kidney product intended for end-stage renal disease via xenotransplant. • uniQure received the green light to proceed with the second dose cohort in their Phase 1/2 trial of AMT-162 for SOD1-ALS gene therapy. • Sarepta Therapeutics' Elevidys showed sustained benefit in ambulatory patients with Duchenne muscular dystrophy in Phase 3 trial results.

Liso-cel Demonstrates Significant Response in Relapsed/Refractory Marginal Zone Lymphoma

7 months ago

• Bristol Myers Squibb's liso-cel achieved a statistically significant and clinically meaningful overall response rate in patients with relapsed or refractory marginal zone lymphoma. • The Phase 2 trial results highlight the potential of liso-cel as an effective treatment option for this challenging patient population. • The data were presented at the 2025 Tandem Meetings, showcasing advancements in cell therapy for hematological malignancies.

Genethon's AAV Gene Therapy GNT0004 Shows Promise in Duchenne Muscular Dystrophy Trial

a year ago

• Genethon's GNT0004 gene therapy demonstrated good tolerability in boys with Duchenne muscular dystrophy when combined with transient immunological prophylactic treatment. • The trial data revealed up to 85 percent of muscle fibers expressing microdystrophin eight weeks post-injection in patients receiving the higher dose. • Patients treated with GNT0004 experienced a decrease in CPK levels, a biomarker of muscular distress, between 50 percent and 87 percent. • One-year efficacy results for the first patient in cohort 2 showed a positive clinical evolution, supporting further pivotal phase development in Europe.

Advancements in AAV Gene Therapy for Duchenne Muscular Dystrophy Show Promise

3 years ago

• AAV-mediated gene therapy is emerging as a promising strategy for treating Duchenne Muscular Dystrophy (DMD) by restoring dystrophin expression. • Micro-dystrophin gene transfer using AAV vectors has demonstrated therapeutic success in large animal models, paving the way for human clinical trials. • Clinical trials by Sarepta Therapeutics, Pfizer, and Solid Biosciences are underway, utilizing different mini-/micro-dystrophin constructs delivered via AAVs. • Managing immune responses and optimizing vector dosage remain key challenges in achieving effective and long-term AAV gene therapy for DMD patients.