Gene Therapies
ARM’s Cell and Gene Meeting on the Mesa offered optimism that a biopharma financial market recovery is under way, but cell and gene therapies still are seen as risky investments.
Big pharma executives at ARM’s Cell and Gene Meeting on the Mesa shared some of the practical challenges of bringing one-time treatments to market in hemophilia, cancer and beyond.
With more advanced therapies gaining approval, companies at ARM’s Cell and Gene Meeting on the Mesa considered hurdles to making their products more broadly available and developing them for larger indications.
Purespring has just raised $105m from an investor syndicate who are betting it can develop the first gene therapy for kidney disease and learn from setbacks experienced in the space.
An upfront payment of $110m from BMS has come just in time for Prime Medicine, but its real test will be the first Phase I data, expected early next year.
The company said the results did not meet the threshold for continued capital investment, giving a boost to Spruce Biosciences and its competing congenital adrenal hyperplasia program.
CorrectSequence led the pack of Chinese cell and gene therapy developers seeking new funding with a roughly $14m series A-plus round. In other modalities, antibody-focused developers Mabgeek and Novamab closed series Bs.
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.
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.
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.
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.
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.