Gene Therapies
The company posted positive initial results from the Heart-2 trial in HeFH and CAD, with strong LDL cholesterol lowering and a clean safety profile.
Adstiladrin sales hit €70m in first full year on the market
Bharat Biotech is the latest entrant in India’s cell and gene therapy space, joining majors like Intas Pharma, Sun Pharma, Cipla and Dr. Reddy’s. An indigenously developed CAR-T cell therapy launched last year, what is driving growing interest and success in cell and gene therapies?
Deal Snapshot: Lilly is the third company to sign a licensing deal for STAC-BBB with Sangamo, which also aims to secure a deal for its Fabry disease program in the second quarter.
The company closed a $150m series C funding round shortly after opening the second part of its Phase I/II study of ATSN-201 and partnering with a Nippon Shinyaku on ATSN-101.
The promise of innovative therapies seems to have been constrained not by efficacy or safety concerns, but because the high price of treatments is incongruous with the reimbursement of short-course therapies.
The founder and CEO of Ultragenyx talked with Scrip as the company approaches a pivotal data readout and awaits US FDA action on its first gene therapy.
Encouraging results in younger children with Duchenne is keeping Regenxbio on track for a potential mid-2026 filing for its gene therapy, RGX-202.
Amgen’s AI model to predict proteins in the “bright Goldilocks zone of viscosity” with over 80% accuracy minus a wet lab experiment holds immense potential, VP Research Haldar said at an event as he spoke about a “hinge moment” for AI in pharma
The gene therapy for Duchenne muscular dystrophy passed the blockbuster sales threshold in 2024. Analysts said the drug’s overall risk/benefit profile still appears solid.
A joint venture with AI specialist Hologen will bring in much-needed cash for the ambitious gene therapy firm.
The company announced early proof-of-concept data for BEAM-302, its base-editing medicine for the genetic liver and lung disease alpha-1 antitrypsin deficiency, showing a clean safety profile.
Cross-Asian initiative has already brought several South Korean gene and cell therapy startups and Japanese VCs closer, with further hopes for product development and launches in Japan, regulatory harmonization and gliobalization.
Policy advances, measures to back local production and crowdfunding action for gene therapies like Zolgensma bring hope for rare disease patients in India. Gaps in financing, high prices and an ongoing court case, however, stunt efforts to ensure timely access.
The drug maker presented data from a Phase I/II trial of DB-OTO that it plans to use to seek regulatory approval.
Pfizer cited many reasons for ending sales of hemophilia B gene therapy Beqvez, including a lack of patient and doctor interest. The company no longer plans to develop AAV gene therapies.
The gene therapy-focused biotech has entered into a deal for private equity firms Carlyle and SK Capital to acquire it amid a shortage of capital and regulatory setback.
Over 140 biopharma leaders share their views on developments to watch for in key therapeutic areas this year. Advances in multiple scientific fields are opening up new avenues for treatment.
Nippon Shinyaku has revised upward its US sales forecast for DMD drug Viltepso, which has overcome multiple challenges in building its position in the US.
Cancer retained its crown as 37 novel drugs were approved for pan-EU marketing last year; treatments for blood disorders followed closely behind in what was another slow year at the European Medicines Agency.