Advanced Therapies
Encouraging results in younger children with Duchenne is keeping Regenxbio on track for a potential mid-2026 filing for its gene therapy, RGX-202.
Miltenyi’s leadership calls for a streamlined regulatory framework as India gears to scale in cell and gene therapy. Executives from the German group also talked to Scrip about helping advance local capabilities, delivering point-of-care CAR- T cell therapy to hospitals and pricing dynamics.
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.
The biotech announced positive Phase III data for Orca-T, its allogeneic cell therapy, and a company cofounder talked with Scrip about Orca’s commercialization plans.
The acquisition is worth up to $1bn and adds to AstraZeneca’s broad array of cell therapy technologies.
A joint venture with AI specialist Hologen will bring in much-needed cash for the ambitious gene therapy firm.
The big pharma will spend $286m to acquire the biotech, which restructured in January 2024 to focus exclusively on their shared product, the CAR-T Abecma.
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 RNAi specialist outlined plans to initiate three Phase III trials this year – two for nucresiran in ATTR amyloidosis and one for zilebesiran for hypertension – during an R&D day in New York.
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.
Biogen will pay $165m up front for ex-North American rights to Stoke’s Phase III-ready Dravet syndrome candidate, an antisense drug offering disease-modifying potential.
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.
Rimqarto is poised to become the first homegrown South Korean CAR-T therapy to gain domestic approval and looks set to provide new local competition to Kymriah.
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.