Advanced Therapies
More than 100 biopharma executives and experts told Scrip their predictions for therapeutic area advances in the coming year. The recent commercial success of GLP-1s in diabetes and obesity and their potential in further disease areas fuelled excitement around the metabolic space. Expectations were also positive in neurology following the launch of Leqembi for Alzheimer’s disease in 2023, while the multiple opportunities to improve cancer treatment kept oncology top of the pile overall.
After 10 years of promised investment following its Nobel Prize for iPS cell research, Japan is cautiously narrowing regulations around the conditional approval of cell therapies and cutting some reimbursement prices. Commercial success remains mixed and some products have been withdrawn from the market.
In this week's podcast edition of Five Must-Know Things: Merck & Co. steps into CD19 bispecific space; gene therapy patients rise, but slowly; Madrigal’s Rezdiffra plans; Korean biopharma financing recovering?; and approvals to watch out for in Q3.
Bluebird, Vertex and Sarepta are starting to see some commercial gene therapy traction and expect momentum to pick up in the second half, while hemophilia remains a challenging area.
After a quiet July, August and September are shaping up to be busy on the approvals front. Already this month, the US FDA has approved Adaptimmune’s Teclera for synovial sarcoma, Phathom’s Voquezna for gastro-esophageal reflux disease and Servier’s Voranigo for gliomas. Here, Scrip takes a look at ten other approvals for novel products in the offing for the third quarter.
The company will cut 65% of its workforce to extend its cash runway and focus its resources on AMT-130 for Huntington's disease and three earlier-stage programs moving into Phase I/II studies.
The company is hoping to become second to market with a DMD gene therapy after Sarepta’s Elevidys, which recently had a label expansion.
Despite initial promise, a novel autologous cell transplant therapy for severe heart failure has faced multiple challenges in gaining full approval in Japan, potentially providing learnings for other developers of regenerative medicines.
The company’s giroctocogene fitelparvovec, partnered with Sangamo, could compete with BioMarin’s hemophilia A gene therapy Roctavian, but longer-term data may be needed.
The two biotechs each hope to offer an Eylea-sparing regimen to wet age-related macular degeneration patients with gene therapies that induce natural production of aflibercept.
The company’s shares fell amid debate over improvements in LVMI, but an analyst pointed out that they indicated the gene therapy has efficacy in Friedreich ataxia cardiomyopathy.
The biotech announced updated interim Phase I/II data for its gene therapy AMT-130, showing statistically significant improvement compared with an external control.
In a major boost for the UK biotech sector, an impressive sum has been raised by Beacon Therapeutics to support its R&D activities in ophthalmic gene therapies.
If finally fully approved in its first indication, the HGF gene therapy could become a first-in-class therapy for PAD - but only after years of challenges in clinical trials.
In this week's podcast edition of Five Must-Know Things: global CEO remuneration; another late-stage loss for Merch KgaA; deals shift to smaller M&A, partnering; Bayer’s Parkinson’s bets bear fruit; and India looks to innovate CAR-T models.
The company presented data showing it is possible to safely and effectively re-dose its ATTR amyloidosis CRISPR/Cas9-based therapy.