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IDefine and UT Southwestern will assess a preclinical EHMT1 gene replacement strategy for Kleefstra syndrome.

Lilly adds Ascidian’s RNA exon-editing platform for rare inherited kidney disease targets in a deal with no disclosed clinical data.

Interim phase 1b/2 data from Tenaya Therapeutics’ MyPEAK-1 study showed TN-201 gene therapy was generally well-tolerated and associated with improvements in cardiac remodeling, symptom burden, and functional measures in patients with MYBPC3-associated hypertrophic cardiomyopathy.¹

Today’s BioPharm Brief covers FDA draft guidance aimed at streamlining rare disease gene therapy development, Phase 3 trial updates involving dual survival endpoints in oncology programs, and Eli Lilly’s $1.2 billion licensing deal for a GLP-2 therapy in short bowel syndrome.

Eli Lilly has entered a licensing agreement with Hanmi Pharmaceutical for sonefpeglutide, a long-acting GLP-2 receptor agonist in Phase 2 development for short bowel syndrome. The deal includes $75 million upfront and up to $1.2 billion in milestone payments, plus royalties.

FDA draft guidance outlines how prior scientific knowledge may support gene therapy development for rare and life-threatening diseases.

Flexible laboratory and good manufacturing practice-ready infrastructure in Basel is designed to support biopharma startups, manufacturing scale-up, and innovation growth, says SENN Development CEO Dr. Johannes Eisenhut.

From multifunctional antibodies and T-cell engagers to in vivo cell therapies and hepatitis B functional cures, the most important large-molecule therapies of 2026 reveal the technological trends driving the next generation of biologic medicines.

Dual primary endpoints were met in both PANKU-Breast02 and PANKU-Esophagus01 phase 3 trials based on interim analyses, while priority review is underway for the esophageal cancer indication in China, Bristol Myers Squibb and Biokin stated.

BioNTech and Bristol Myers Squibb report confirmed ORRs exceeding 60% at the lower dose in both non-squamous and squamous first-line NSCLC; three global Phase 3 trials are now enrolling.

New clinical and translational data presented at ASCO 2026 and published in Nature Medicine demonstrate encouraging anti-tumor activity and manageable safety for Immatics’ IMA401 TCR bispecific in patients with MAGE-A4-positive solid tumors.

Pfizer’s $10.5 billion oncology collaboration with Innovent, early clinical progress for a novel PD-1/IL-2 fusion protein, and promising Phase III hepatitis B cure data highlight today’s key developments in biopharmaceutical innovation.