Genetic Function
Epigenic Therapeutics and NRG Therapeutics Each Secure ~$60M in Series B Rounds for Epigenetic and Neuroscience Advances
Epigenic Therapeutics; NRG Therapeutics; Series B funding; epigenetic therapy; hepatitis B; hypercholesterolemia; Parkinson’s disease; ALS; biotechnology investment; gene silencing
Wave RNA Editing Restores Enzyme in Genetic Disorder but Fails to Impress Investors
Wave Life Sciences; RNA editing; AATD; alpha-1 antitrypsin deficiency; enzyme restoration; clinical trial; investor reaction; WVE-006
Wave Life Sciences Shares Dip After Mixed Data Update on GSK-Partnered AATD Therapy
Wave Life Sciences; GSK; AATD; RNA editing; WVE-006; clinical trial; protein levels; stock dip; biotech
Novartis Again Partners with Argo Biopharma in $5.2B+ RNAi Cardiovascular Deal
Novartis; Argo Biopharma; RNA interference (RNAi); siRNA; cardiovascular pipeline; ANGPTL3; dyslipidemia; biotech collaboration; drug development; milestone payments
Novartis Takes Another Shot at Alpha-Synuclein With $2.2B Arrowhead CNS Deal
Novartis; Arrowhead Pharmaceuticals; alpha-synuclein; Parkinson’s disease; RNA interference (RNAi); gene silencing; ARO-SNCA; TRiM platform; neurodegeneration; biotech partnerships
Roche and Alnylam Push Zilebesiran into Phase III Trial Despite Mixed Phase II Hypertension Data
Roche; Alnylam; zilebesiran; hypertension; RNAi therapeutics; KARDIA-3; Phase II trial; Phase III trial; cardiovascular outcomes; ZENITH trial
Merck KGaA Forms $2 Billion Neurological Disease Pact with Skyhawk Therapeutics
Merck KGaA; Skyhawk Therapeutics; neurological diseases; RNA splicing; SkySTAR platform; drug discovery; strategic collaboration; milestone payments
Sarepta Sells $174 Million Stake in Arrowhead to Fund Milestone Payment Amid Cash Crunch
Sarepta Therapeutics; Arrowhead Pharmaceuticals; cash crunch; stake sale; milestone payment; RNAi collaboration; SRP-1003; myotonic dystrophy; private block trade
Boehringer Ingelheim breaks into oncology as FDA grants accelerated approval to Hernexeos (zongertinib) for HER2 TKD–mutated NSCLC
Boehringer Ingelheim; Hernexeos; zongertinib; FDA approval; accelerated approval; non-squamous NSCLC; HER2 TKD mutations; Oncomine Dx Target Test; Beamion LUNG-1; objective response rate; first oral targeted therapy for HER2-mutant NSCLC; priority review; breakthrough therapy designation; confirmatory trial requirement; competition with Enhertu
Sanofi Acquires China Rights to Arrowhead’s Plozasiran for $395 Million
Sanofi; Arrowhead Pharmaceuticals; Visirna Therapeutics; Plozasiran; China rights; RNA interference; Familial chylomicronemia syndrome; Severe hypertriglyceridemia; biotech partnership; milestone payments