rare disease

As recently as December 10, 2025, Rezolute was a Wall Street darling. Eight analysts rated the stock a Buy, with price targets as high as $17. Its lead drug ersodetug, a monoclonal antibody, had earned two FDA Breakthrough Therapy Designations and the agency had signaled that its Phase 3 trial could support a BLA filing.…

The American Society of Gene & Cell Therapy (ASGCT) and Orphan Therapeutics Accelerator (OTXL), a biotech non-profit, have launched the CGTxchange, a jointly owned clearinghouse and marketplace designed to identify, evaluate and advance clinically validated cell and gene therapies (CGTs) for ultra-rare diseases that have been shelved due to commercial constraints.  This will serve as…

Novo Nordisk’s $5.2 billion acquisition of Akero Therapeutics sits at the center of 2025’s hottest M&A space: MASH/metabolic disease. The deal, announced October 9, represents the largest of three major Fibroblast Growth Factor 21 (FGF21) analog acquisitions in five months. Across the broader landscape, 2025 pharma M&A activity reached approximately $70 billion in upfront consideration…

Despite challenging interest rates and regulatory environment, pharma M&A activity surged in 2023 with deal volume up more than 30% from the prior year based on a review of more than 200 acquisitions since 2018. The total disclosed deal value in 2023 also more than doubled the prior year’s tally to surpass $100 billion. While…

Many large drug developers employ individual groups or teams independently working on discrete steps in the drug development process. Rare disease biopharma Insmed (NSDQ:INSM) differentiates itself through its focus on patients with rare diseases. To that end, the company works to ensure collaboration between various departments to consider questions related to the applicability of a…