Entrada Therapeutics

Vertex Pharmaceuticals Abandons AAV Vector Technology Amid Broader Industry Retreat from Gene Therapy

15 days ago

• Vertex Pharmaceuticals has discontinued all research on adeno-associated virus (AAV) vector technology, impacting partnerships with Affinia Therapeutics and Tevard Biosciences focused on Duchenne muscular dystrophy treatments. • The retreat from AAV vectors follows a broader industry trend, with Pfizer, Roche, Takeda, and Biogen all scaling back gene therapy programs due to safety concerns, limited payload capacity, and high manufacturing costs. • Despite industry pullback, companies like Affinia Therapeutics continue developing next-generation AAV vectors, while others explore alternative delivery systems such as Herpes simplex virus-1 vectors with larger genetic payloads.

FDA Grants RMAT Designation to Elixirgen's Novel Gene Therapy for Rare Telomere Disorders

3 months ago

• Elixirgen Therapeutics' gene therapy EXG-34217 receives FDA's Regenerative Medicine Advanced Therapy designation for treating Telomere Biology Disorders with bone marrow failure. • The therapy, currently in Phase 1/2 trials, uses modified hematopoietic stem cells expressing ZSCAN4 protein to address telomere elongation in patients with these rare genetic conditions. • RMAT designation provides accelerated development and review pathways, including Fast Track and Breakthrough Therapy benefits, for this treatment targeting disorders affecting 1 in 1 million Americans.

Entrada Therapeutics Advances DMD Treatment: UK MHRA Approves Phase I/II Trial for Novel Exon-Skipping Therapy

4 months ago

• Entrada Therapeutics has received MHRA authorization to commence ELEVATE-44-201, a Phase I/II trial evaluating ENTR-601-44 for Duchenne muscular dystrophy patients with exon 44 skipping mutations. • The global trial consists of two parts: a multiple ascending dose study with 24 subjects receiving 6-18mg/kg doses, followed by an efficacy and safety assessment phase with quality-of-life measures. • ENTR-601-44, an Endosomal Escape Vehicle-conjugated therapy, aims to restore functional dystrophin protein production, with trial initiation planned for Q2 2024.

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.

Medtronic's BrainSense Adaptive DBS Receives CE Mark for Personalized Parkinson's Therapy

4 months ago

• Medtronic's BrainSense Adaptive Deep Brain Stimulation (aDBS) and Electrode Identifier (EI) have received CE Mark approval in the EU and UK. • The aDBS system offers real-time, self-adjusting brain stimulation for Parkinson's disease, adapting to individual patient's unique brain activity. • BrainSense Electrode Identifier improves DBS programming by facilitating optimal initial contact selection, reducing programming time for clinicians. • The first European patient has already been programmed with the new system at Amsterdam University Medical Center, marking a significant advancement.

Biopharma Industry Faces Continued Layoffs in 2025 Amid Strategic Realignments

4 months ago

• Biopharma companies continue to announce layoffs in 2025, driven by strategic realignments, pipeline reprioritizations, and challenging financial conditions. • Several companies, including Intellia Therapeutics and IGM Biosciences, are cutting a significant portion of their workforce to focus on key programs and extend cash runways. • These workforce reductions are often accompanied by discontinuation of certain drug development programs and a shift in strategic focus towards core assets. • The layoffs reflect a broader trend in the industry as companies adapt to evolving market dynamics and prioritize efficiency and long-term sustainability.

Glucarpidase Improves Kidney Recovery in Methotrexate-Associated Acute Kidney Injury

5 months ago

• A recent study found that glucarpidase treatment significantly improves kidney recovery in patients with methotrexate-associated acute kidney injury (AKI). • Patients receiving glucarpidase showed a 2.7-fold higher odds of kidney recovery compared to those without glucarpidase, according to the study. • Glucarpidase treatment was also associated with faster time-to-kidney recovery and reduced risks of neutropenia and transaminitis. • The findings support the use of glucarpidase to improve renal and extrarenal outcomes in adults experiencing MTX-AKI.

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.

FDA Approves Supernus' ONAPGO for Motor Fluctuations in Advanced Parkinson's Disease

7 months ago

• The FDA has approved ONAPGO (apomorphine hydrochloride) as the first subcutaneous apomorphine infusion device for treating motor fluctuations in advanced Parkinson's disease. • ONAPGO, a wearable device, provides continuous subcutaneous apomorphine infusion, offering more consistent control of OFF time for Parkinson's patients. • Clinical trials demonstrated that ONAPGO significantly reduced daily OFF time by 2.6 hours compared to placebo, with improvements seen as early as week 1. • Supernus plans to launch ONAPGO in the U.S. in the second quarter of 2025, supported by a dedicated nurse education program and access support.