Gene Therapies
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.
After ditching its clinical-stage ex vivo candidate in December, the CRISPR-based company is starting all over again with a focus on in vivo therapies and upregulating beneficial genes.
The Swiss giant’s blockbuster gene therapy is currently only administered intravenously into patients with spinal muscular atrophy under two years of age. Its intrathecal candidate, called OAV-101, has just passed a Phase III test which could expand its use in children and young adults.
China's Belief BioMed presented promising new Phase III results at ASH for its AAV gene therapy for hemophilia B, although the wider China CGT space continues to be dominated by oncology.
UniQure said discussions with the FDA resulted in a path forward for seeking accelerated approval of its gene therapy AMT-130 in Huntington’s disease.
The Swiss pharma acquired Kate Therapeutics for up to $1.1bn, gaining preclinical gene therapies for neuromuscular conditions and novel platform technology.