Biotech & Research

SHERIDAN, WYOMING – October 15, 2025 – Sarepta Therapeutics is pressing forward with its limb-girdle muscular dystrophy (LGMD) gene therapy, SRP-9003, despite a strategic pivot earlier this year toward small interfering RNA (siRNA) therapies and ongoing regulatory challenges. New late-stage data presented at the World Muscle Society Congress in Vienna suggest the therapy could still hold clinical and commercial potential—if Sarepta can navigate FDA scrutiny and rebuild trust in its gene therapy platform.

SHERIDAN, WYOMING – October 15, 2025 – Pelage Pharmaceuticals has raised $120 million in Series B financing to advance its novel stem cell–activating therapy for hair loss into Phase III clinical trials. The funding positions the California-based biotech to accelerate clinical development of PP405, a first-in-class topical molecule designed to rejuvenate dormant hair follicle stem cells and restore natural hair growth.

SHERIDAN, WYOMING – October 15, 2025 – Boehringer Ingelheim is continuing its high-stakes investment strategy in next-generation oncology treatments, signing a deal worth up to $991 million with South Korea’s AimedBio to license an antibody-drug conjugate (ADC) for cancer. The move underscores Boehringer’s accelerating focus on precision oncology and reinforces its growing footprint in the ADC space—one of the most competitive frontiers in cancer therapeutics.

SHERIDAN, WYOMING – October 15, 2025 – Bristol Myers Squibb (BMS) has expanded its artificial intelligence-driven collaboration with insitro, reinforcing its long-term strategy to accelerate therapeutic development in neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS). The renewed deal underscores a shared commitment to leveraging AI and advanced chemistry platforms to unlock new pathways in ALS drug discovery.

AI-Powered Extension Strengthens a Five-Year Alliance

SHERIDAN, WYOMING – October 15, 2025 – BioCryst Pharmaceuticals is strengthening its hereditary angioedema (HAE) pipeline through the acquisition of Massachusetts-based Astria Therapeutics in a $700 million cash-and-stock deal. The move positions BioCryst to compete more aggressively in the rare disease market by combining its oral HAE treatment, Orladeyo, with Astria’s long-acting injectable candidate, navenibart.

SHERIDAN, WYOMING – October 15, 2025 – In a pivotal development for the U.S. biotech sector, Krystal Biotech has received the U.S. Food and Drug Administration’s (FDA) second-ever platform technology designation for its non-replicating HSV-1 viral vector—positioning the company as a key early beneficiary of the agency’s emerging regulatory framework for gene therapy platforms.

Validation of a Versatile Gene Delivery Platform