Gene Therapies

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.

The German major is kicking off Phase II trials for a cell therapy and a gene therapy that could be gamechangers for the progressive neurodegenerative disorder that affects more than 10 million people worldwide.

SanBio’s lead cell therapy asset has been on a bumpy journey to its global-first approval and while a nod has now come in Japan in a high-need indication, a commercial launch is conditional on additional data to establish product equivalence and manufacturing consistency. 

As India seeks to build on early gains in CAR-T cell therapy, experts call for ecosystem reform and also the need to demonstrate value to payer models of large economies to potentially back international reimbursements.

The gene therapy is now approved for all ambulatory boys with Duchenne muscular dystrophy ages 4 and older. The revised label also includes accelerated approval for non-ambulatory patients.

A first big pharma partnership for Ascidian, headed up by ex-Biogen research head Michael Ehlers, as the Swiss firm expands its gene editing and neuroscience efforts.

The merger of Freeline and SwanBio is establishing an entity with secure financing to advance their respective gene therapies for Gaucher’s disease and adrenomyeloneuropathy.

Pfizer’s Phase III CIFFREO study in DMD failed to meet primary and key secondary endpoints, likely removes it as a potential competitor to Sarepta’s Elevidys.

With several new drugs progressing for the estimated 70,000 patients in China with DMD, a recent setback for Nippon Shinyaku's contender and progress for Chinese gene therapy developers are adding both uncertainty and likely competition in an untapped market with pressing medical need.