Hereditary Diseases
BioMarin’s R&D Chief Warns of Innovation Slowdown Amid NIH Funding Cuts
BioMarin, NIH funding, biomedical innovation, genetic diseases, drug development, research partnerships
BridgeBio Pharma Reports Strong Commercial Progress and Pipeline Updates for 2025
BridgeBio Pharma, Attruby, commercial progress, pipeline updates, 2025 milestones, rare genetic diseases, Phase 3 clinical trials, FORTIFY, CALIBRATE, PROPEL 3.
FDA Approves Neurocrine’s Crenessity for Rare Congenital Adrenal Hyperplasia
Neurocrine Biosciences, FDA approval, Crenessity, crinecerfont, congenital adrenal hyperplasia (CAH), rare genetic disease, adrenal gland condition.
Sarepta Therapeutics and Arrowhead Pharmaceuticals Enter $825M Global Licensing and Collaboration Agreement for Multiple siRNA Programs
Sarepta Therapeutics, Arrowhead Pharmaceuticals, siRNA programs, Global Licensing and Collaboration Agreement, Rare genetic diseases, Gene therapy, RNA interference
FDA Approves PTC Therapeutics’ Kebilidi, the First Gene Therapy Delivered Directly to the Brain for AADC Deficiency
PTC Therapeutics, Kebilidi, Gene Therapy, AADC Deficiency, FDA Approval, Brain-Delivered Therapy, Rare Genetic Disorder
Revolutionizing AATD Treatment: Emerging Therapies to Replace Augmentation Therapy
Alpha-1 Antitrypsin Deficiency (AATD), Genetic Disorder, New Therapies, Augmentation Therapy, RNA Interference (RNAi), Gene Editing, CRISPR
Wave Life Sciences Sees Historic Stock Surge Following Breakthrough in RNA Editing Therapy
Wave Life Sciences, RNA editing, genetic condition, lung and liver complications, WVE-006, biotech, stock surge, clinical trial data
BioMarin and CAMP4 Collaborate on RNA-Targeting Therapies for Rare Genetic Diseases
BioMarin, CAMP4, RNA-targeting therapies, genetic diseases, regulatory RNA, RAP Platform, antisense oligonucleotides, ASOs, rare genetic conditions
Aptadir Therapeutics Unveils Novel RNA Inhibitors to Combat Cancer and Genetic Disorders
Aptadir Therapeutics, RNA inhibitors, DNMT1 interacting RNAs (DiRs), Cancer treatment, Genetic diseases, Epigenetic targeting, RNA therapeutics
Novo Nordisk Partners with NanoVation in $600M Deal for Advanced Genetic Medicine Delivery
Novo Nordisk, NanoVation Therapeutics, Genetic Medicine, Lipid Nanoparticle (LNP) Technology, Cardiometabolic Diseases, Rare Genetic Disorders