Gene therapies for Fabry and Gaucher disease showed sustained benefits in early trials: all 11 Fabry patients stopped enzyme replacement therapy, and four of six Gaucher patients stayed off standard treatment for up to two years.
Keros Therapeutics plans a phase II DMD trial of rinvatercept in Q1 2026 and regulatory talks for ALS in H2 2026. Cash stands at $383 million with runway into H1 2028.
Genetic doping is no longer theoretical; 92% of athletes underwent anti-doping testing before the Winter Olympics 2026. Experts propose genomic passports, noting no confirmed cases but black-market availability of plasmid-based EPO genes.
Novartis marks 30 years since its 1996 merger, citing advances in targeted therapy, cell therapy, gene therapy, and malaria. It also named a Levant Country President and won two clinical research awards in Saudi Arabia.
Biotech companies posted Q2 2026 results: REGENXBIO's Duchenne trial met its primary endpoint, Compass Pathways' COMP360 NDA submission is on track for Q4, Puma raised full-year guidance on NERLYNX demand, and Evotec cut its 2026 outlook.
Life Biosciences has treated the first patient with ER-100, a cell-rejuvenating gene therapy for glaucoma. The FDA-approved Phase 1 trial will assess safety in up to 18 patients.
Genomic medicine market is projected to reach USD 123 billion by 2035; AI in genomics USD 6.5 billion; viral vector manufacturing USD 15.83 billion. Gene therapy trials drive growth.
Novartis beat second-quarter profit expectations as Kisqali, Kesimpta and Leqvio offset a 50% plunge in Entresto sales. The Swiss drugmaker maintained its full-year guidance and awaits late-stage data on three experimental drugs.
A stem cell therapy for geographic atrophy improved vision in a phase Ib trial, and the first gene therapy for optic neuropathies was administered. Next-generation anti-VEGF agents are also extending treatment intervals in retinal vascular disease.
England will screen all newborns for SMA after Jesy Nelson's campaign. A real-world study found Zolgensma helped children with SMA achieve motor milestones, with newborn screening leading to earlier gains.
