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May 20, 2026
The FDA fast-tracked an inhalable gene therapy for advanced lung cancer after an early 11-patient trial showed tumour shrinkage in three patients and disease stabilization in five. Larger studies involving about 250 patients are underway.
May 20, 2026
Two Louisiana parents published a children’s book to raise awareness of Infantile Neuroaxonal Dystrophy, a rare disorder affecting two of their daughters. They are also helping raise money for a gene therapy trial.
May 19, 2026
Sensorion appointed Fred Chereau chief executive effective June 1, 2026, while reporting six-month Audiogene trial data for SENS-501. The company said early hearing improvements in cohort 2 persisted at six months and no serious adverse events were reported.
May 16, 2026
Benitec Biopharma's gene therapy BB-301 improved swallowing ability in all four OPMD patients followed for at least one year in a Phase Ib/II trial. One patient showed continued improvement at two years. The company plans to meet with the FDA in mid-2026 to discuss a pivotal study.
May 14, 2026
A U.S. appeals court revived Regenxbio’s patent suit against Sarepta, holding the asserted gene-therapy host cell claims are markedly different from anything occurring in nature.
May 12, 2026
Phase 1 data in 17 adults with Friedreich's ataxia cardiomyopathy showed AAVrh.10hFXN (LX2006) was generally well tolerated and showed signs of biological activity. The gene therapy is being advanced into a larger trial.
May 12, 2026
Recent studies in retinitis pigmentosa showed mixed results for gene and optogenetic therapies. UGX-201 and MCO-010 were associated with visual acuity improvements, while AAV8.hPDE6A showed no significant functional gains at 1 year.
May 10, 2026
Vertex secured a Germany reimbursement agreement for CASGEVY and signed a WuXi Biologics pact for a preclinical autoimmune T-cell engager. The moves add to efforts to build revenue beyond cystic fibrosis.
May 05, 2026
India is expanding rare disease support under the 2021 policy, with aid up to Rs 50 lakh and 15 Centres of Excellence. Officials also called for local innovation as treatment costs can reach ₹16 crore.
May 05, 2026
The United Arab Emirates has unveiled two gene therapy projects targeting autism, epilepsy, and rare brain disorders in children. The initiatives focus on underlying genetic causes and earlier diagnosis.
May 01, 2026
Ray Therapeutics received EMA PRIME designation for its RTx-015 optogenetic gene therapy for retinitis pigmentosa, following recent FDA RMAT status. The company also appointed Michael Murtagh as Chief Regulatory Officer to support global development.
Apr 21, 2026
The FDA said uniQure’s Phase I/II data for Huntington’s disease gene therapy AMT-130 are not adequate for a marketing application. The agency recommended a new randomised, double-blind, sham surgery-controlled study.
Apr 19, 2026
Rznomics said it will present interim AACR results for liver cancer candidate RZ-001 from a U.S. trial in patients with hepatocellular carcinoma. Tumor-shrinking responses were observed, and about 23% experienced complete disappearance of cancer cells.
Apr 18, 2026
Novartis is emphasizing innovative medicines across oncology, immunology, neuroscience and gene therapy. The company targets mid-single-digit sales growth through 2027 and core operating income margins above 35%.
Apr 18, 2026
A lipid nanoparticle gene-editing approach inserted a full healthy CFTR gene into human airway cells and restored 88% to 100% of normal CFTR function in lab tests. The nonviral strategy is intended as a mutation-agnostic path for cystic fibrosis.
Apr 11, 2026
Pharma robots and clean room pass-through systems are projected to grow through 2035 as drugmakers increase automation and contamination control. Demand is being driven by biologics, cell and gene therapies, stricter sterile-manufacturing requirements, and investment in cleanroom infrastructure.
Apr 08, 2026
Duchenne muscular dystrophy care is shifting toward broader multidisciplinary management and closer attention to early pathology, biomarkers, and treatment timing. Clinicians described an expanding treatment landscape and identified neurology, cardiology, pulmonary, and endocrinology as core priorities.
Apr 06, 2026
The microfluidics market is projected to grow from $10.33 billion in 2025 to $20.73 billion by 2030. Growth is tied to lab-on-a-chip research, miniaturized diagnostics, and pharmaceutical R&D adoption.
Apr 04, 2026
Krystal Biotech is set to report fourth-quarter results with analysts expecting $1.58 a share on $106.16 million in revenue. Investors are watching Vyjuvek growth and pipeline programs including KB801, KB407 and KB707.
Apr 03, 2026
Ocugen has completed enrollment and dosing in its phase II/III GARDian3 study for OCU410ST gene therapy targeting Stargardt disease. The study includes 63 patients and aims to address over 1,200 ABCA4 gene mutations with a single treatment. Interim data is expected in Q3 2026, with a BLA submission targeted for mid-2027.
