Can Greenstone Biosciences turn patient-derived heart cells into a Duchenne cardiomyopathy drug?
Greenstone Biosciences secured an NIH R61 grant to discover cardiac fibrosis drugs for Duchenne muscular dystrophy using iPSC and AI models.
Pharma & Biotech
Pharma & Biotech
Beam Therapeutics is trying to rewrite a disease-causing mutation inside the liver. Its pivotal test has now begun
Beam Therapeutics has begun dosing in the global pivotal cohort of its BEAM-302 trial, advancing a one-time in vivo base-editing therapy that could address both lung and liver disease in people with severe alpha-1 antitrypsin deficiency.
Pharma & Biotech
Rhythm’s weekly obesity drug matched early results from daily treatment, but only seven patients reached the key assessment
Rhythm Pharmaceuticals has reported preliminary RM-718 data showing an 11.6% mean body mass index reduction after 16 weeks in seven patients with acquired hypothalamic obesity. The small, open-label dataset supports further development but cannot yet establish how the weekly injection compares with approved daily setmelanotide.
Pharma & Biotech
Neurocrine NBIP-1968 enters Phase 1 as triple-agonist obesity race accelerates
Neurocrine Biosciences has initiated a first-in-human Phase 1 study of NBIP-1968, a once-weekly experimental obesity treatment designed to activate the GLP-1, GIP and glucagon receptors through a single molecule. The August 7, 2026 development marks Neurocrine Biosciences’ entry into clinical testing with an incretin-based obesity candidate. The study will initially examine single ascending doses in
Pharma & Biotech
Capricor lost the FDA panel vote 9-3. Why investors have not completely abandoned deramiocel
Capricor Therapeutics faces an August 22 FDA decision for deramiocel after advisers voted that the available evidence did not establish effectiveness in Duchenne muscular dystrophy cardiomyopathy. The disputed case now turns on statistical methods, the treatment indication and whether supportive upper-limb results can overcome unresolved cardiac evidence.
Pharma & Biotech
Replimune survived two FDA rejections and won the panel vote. Why RP1’s hardest question remains unanswered
Replimune Group is awaiting a delayed FDA decision on RP1 after advisers voted that its melanoma results were clinically meaningful. Approval would create a more accessible oncolytic immunotherapy option, but regulators remain divided over whether the single-arm IGNYTE trial reliably proves RP1’s contribution.
Pharma & Biotech
Can one injection challenge daily hot-flash drugs? AbCellera’s ABCL635 faces its first real efficacy test
AbCellera will report Phase 2 ABCL635 data on Aug. 10, testing whether a long-acting NK3 antibody can reduce menopause hot flashes.
Pharma & Biotech
Apitegromab survives another manufacturing hurdle: Can Scholar Rock still secure FDA approval by September 30?
Scholar Rock shifts apitegromab’s FDA review to a second fill-finish site as its September 30 SMA decision date remains on track.
Pharma & Biotech
Faeth Therapeutics has picked its PIKTOR dose. The real test arrives by year-end
Faeth Therapeutics explains the PK/PD rationale behind PIKTOR dosing as its Phase 2 endometrial cancer trial approaches a 2026 readout.
Pharma & Biotech
RiboX RXIM002 FDA clearance takes circular RNA in vivo CAR-T into autoimmune disease trials
RiboX Therapeutics has received clearance from the United States Food and Drug Administration to begin clinical testing of RXIM002, an experimental treatment designed to create temporary anti-CD19 CAR-T cells directly inside the body of a patient with autoimmune disease. The August 8, 2026 investigational new drug clearance allows RiboX Therapeutics to initiate the Phase 1