Disease or Syndrome
Novo throws weight behind budding rare disease pipeline
Novo Nordisk; rare disease; pipeline; Omeros; Zaltenibart; MASP-3 inhibitor; PNH; complement system; milestone payments
Krystal Biotech Receives FDA Platform Technology Designation for HSV-1 Viral Vector Used in KB801 for Neurotrophic Keratitis
Krystal Biotech; FDA; platform technology designation; HSV-1 viral vector; KB801; gene therapy; neurotrophic keratitis; redesable eye drop; VYJUVEK
Novo Nordisk Acquires Rights to Omeros’ MASP-3 Inhibitor Zaltenibart
Novo Nordisk; Omeros; Zaltenibart; MASP-3 Inhibitor; Rare Blood and Kidney Disorders
Gene Therapy Delivers Long-Term Immune Protection in Children With Rare Immune Disorders
gene therapy; long-term immune protection; ADA-SCID; severe combined immunodeficiency; LAD-I; leukocyte adhesion deficiency; rare disease; pediatric immunology; clinical trial; curative treatment
Are the Good Times in Biotech Back? – Insights from Post-Hoc Live’s 2025 Industry Sentiment
biotech sentiment; Post-Hoc Live; 2025 industry outlook; biotech M&A; FDA delays; executive survey; public biotech companies; JP Morgan effect; biotech funding
Lilly’s Oral GLP-1, Orforglipron, Achieves Two New Clinical Wins in Type 2 Diabetes
Lilly; Orforglipron; oral GLP-1; type 2 diabetes; clinical trial; weight loss; regulatory submission; ACHIEVE-1; ATTAIN-2; semaglutide; Farxiga; efficacy; safety
Pelage Pharmaceuticals Raises $120M to Advance Hair Loss Regenerative Therapy to Phase 3 Trials
Pelage Pharmaceuticals; hair loss; regenerative medicine; PP405; Phase 3 clinical trial; Series B financing; stem cell therapy; androgenetic alopecia
BioCryst Snaps Up Fellow Angioedema Player Astria for $700M
BioCryst; Astria Therapeutics; acquisition; hereditary angioedema (HAE); navenibart; rare disease; pharmaceutical industry; cash and stock deal; Jill Milne; $700 million
Lunit AI Predicts Immunotherapy Outcomes in Colorectal, Kidney, and Lung Cancer at ESMO 2025
Lunit AI; SCOPE IO; immunotherapy; colorectal cancer; kidney cancer; lung cancer; biomarkers; ESMO 2025; treatment prediction; AI pathology
Spotlight On: Cabaletta Bio’s Small but Mighty Breakthrough for CAR-T in Autoimmune Arena
Cabaletta Bio; CAR-T therapy; rese-cel; autoimmune diseases; B cell depletion; myositis; systemic lupus erythematosus; systemic sclerosis; pemphigus vulgaris; preconditioning; clinical trial; FDA