Related coverage linked through entity extraction aliases.
Aug 23, 2026
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.
Aug 14, 2026
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.
Aug 12, 2026
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.
Jul 24, 2026
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.
Mar 18, 2026
Candel Therapeutics secured $100M royalty funding for CAN-2409 and plans a Q4 2026 BLA submission in prostate cancer. Updated NSCLC phase 2a data showed median overall survival of 25.4 months, with a phase 3 trial planned for Q2 2026.
Jun 20, 2026
India convened a national conference on rare diseases, Irish lawmakers debated Skyclarys access, and Malta unveiled its first rare disease strategy. New measures include expanded Centres of Excellence, ₹50 lakh assistance, and givinostat reimbursement.
Jul 09, 2026
Biogen marked 30 years of U.S. manufacturing with a $2 billion RTP investment, while regional leaders urged deeper venture capital and research density to boost North Carolina's biotech competitiveness.
Jul 15, 2026
Researchers at MD Anderson developed an extracellular vesicle-based platform that delivers full-length DMD mRNA, restoring dystrophin production and improving muscle function in preclinical Duchenne muscular dystrophy models. The study, published in Nature Biomedical Engineering, showed reduced side effects compared with viral gene therapies.
Aug 09, 2026
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.
Aug 07, 2026
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.
Apr 29, 2026
Global biologics market projected to grow to USD 1.28 trillion by 2035; Japan cell and gene therapy market to reach USD 2.15 billion by 2034; Japan biologic drug lifecycle management market to hit USD 9.69 billion by 2033.
May 24, 2026
Axol Bioscience has acquired the ophthalmology business of Newcells Biotech, adding advanced retinal organoid and 2D RPE models to its iPSC portfolio. The deal follows Axol's acquisition of Phenocell and a $2.8 million financing led by BroadOak Capital Partners. Financial terms were not disclosed.
Jul 31, 2026
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.
May 31, 2026
Positive Phase 3 VOXZOGO results in hypochondroplasia; FDA sNDA submission planned in Q3. Q4 2025 revenue rose 17% to $874.6M, beating estimates; EPS missed. Q1 2026 results due May 4.
Feb 24, 2026
Myriad Genetics, uniQure, and Legend Biotech have scheduled Q4 and full year 2025 earnings calls for Feb 23, Mar 2, and Mar 10, 2026, respectively. Details include webcast access, dial-in numbers, and company background.
Jul 27, 2026
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.
Jul 27, 2026
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.
Jul 18, 2026
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.
Jun 20, 2026
Amgen shares have fallen 12.6% in three months despite relatively solid Q1 results, while Novo Nordisk maintains a 54.6% global GLP-1 market share. Both companies face pipeline competition and pricing pressures.
Jul 17, 2026
A securities class action lawsuit alleges REGENXBIO misled investors about the safety of its gene therapy RGX-111. Following an FDA clinical hold due to a patient tumor, the stock fell 17.8% in one day. Affected shareholders have until April 14, 2026 to seek lead plaintiff status.
