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Antisense oligonucleotides are reshaping drug design, from RNase H gapmers to inhaled delivery; this FAQ explains how they work and why trials still fail.

Bristol Myers Squibb's Nathan Pennell explains how the MRD endpoint supports accelerated approval in multiple myeloma without changing how clinicians monitor patients.

Once-weekly amylin analog petrelintide produced up to 9.2% weight loss at 28 weeks in adults with obesity and type 2 diabetes, Zealand Pharma reports.

Teprotumumab N01 (Sycume) reduced eye bulging in 60.4% of patients with chronic, inactive thyroid eye disease in a phase 3 trial.

Tecentriq plus chemotherapy gains FDA approval for stage III dMMR colon cancer after a phase 3 trial showed improved disease-free survival.

New clinical milestones, manufacturing investments, and regulatory incentives could help developers overcome barriers to delivering treatments for patients with rare diseases.

Genglycos, the gene therapy that earned the voucher, will fund new Ultragenyx therapies for other rare and ultra-rare diseases.

Series A led by Oxford Science Enterprises and up to $20 million in public funding will move cell-based mRNA production toward clinical-grade scale.

Rocket’s modified protocol keeps the first three patients in a 12-patient efficacy population, with topline Danon disease data expected in mid-2028.

Caribou is halting vispa-cel and CB-011, allogeneic CAR-T therapies that showed up to 92% response rates, citing a difficult financing environment.

Celltrion USA's Omlyclo, the first interchangeable biosimilar to Xolair, reaches the U.S. market across four allergic and inflammatory indications.

Alvotech turns to a CDMO's 40,000-liter Syracuse facility for biosimilar drug substance as it expands its manufacturing network.

AbbVie's c-Met ADC Temab-A showed a 15.6% response rate in late-line colorectal cancer ahead of its 2 new FDA breakthrough designations.