Codexis | MedPath

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.

Rivus Pharmaceuticals Strengthens Leadership Team Ahead of Critical Phase 2b MASH Trial Results

a month ago

• Rivus Pharmaceuticals has appointed David Grainger, Ph.D., as Chairman of Development and Meg Fitzgerald, J.D., as Chief Legal Officer while establishing a new office in South San Francisco. • The company is advancing its novel Controlled Metabolic Accelerators (CMAs), led by HU6, which has shown promising Phase 2a results in treating obesity-related conditions without muscle mass loss. • Rivus is approaching a critical milestone with the imminent readout of its Phase 2b M-ACCEL study in metabolic dysfunction-associated steatohepatitis (MASH), potentially advancing HU6 to pivotal trials.

Advancements in Clinical Trials for Fabry Disease, C. difficile Infections, and Mydriasis

6 months ago

• Clinical trials are actively progressing for Fabry Disease, with companies like Idorsia and Sanofi Genzyme developing novel therapies. • Emerging treatments like Venglustat and Pegunigalsidase alfa are expected to significantly impact the Fabry Disease market in the coming years. • Research and development efforts are also focused on Clostridium Difficile Infections, with Vedanta Biosciences leading Phase 3 trials for VE303. • Mydriasis treatments are advancing, with companies like Eyenovia exploring solutions like MYDCOMBI for pupil dilation.

Artiva Biotherapeutics Announces AlloNK® Clinical Updates and Q3 2024 Financial Results

6 months ago

• Artiva Biotherapeutics reported the initiation of AlloNK® (AB-101) treatment in a basket trial for autoimmune diseases like rheumatoid arthritis and lupus, with initial data expected in H1 2025. • The company expanded its Board of Directors with the appointment of Dr. Alison Moore, enhancing cell therapy and manufacturing expertise to advance AlloNK® in autoimmune indications. • Artiva's financial position remains strong with $199.6 million in cash, cash equivalents, and investments, expected to fund operations through the end of 2026. • AlloNK® is undergoing clinical evaluation in combination with rituximab or obinutuzumab in a Phase 1/1b trial for systemic lupus erythematosus, including patients with or without lupus nephritis.