Gene Therapy
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CRISPR eyes autoimmune disease in revamp of cell therapy plans
The gene editing biotech is shelving two of its most advanced cancer drugs, and joining a growing group of companies exploring cell-based medicines for inflammatory diseases like lupus.
By Ben Fidler • Dec. 5, 2023 -
BioMarin secures hemophilia gene therapy coverage in Germany
Drawn-out negotiations led to a lower price than initially expected, but analysts called the agreement a step forward for the biotech company.
By Kristin Jensen • Nov. 29, 2023 -
Explore the Trendline➔
Mario Tama via Getty ImagesTrendlineGene Therapy
Rapid scientific advances have put the gene therapy field at the forefront of biomedical research. But, as recent setbacks have shown, researchers and drugmakers still face major challenges.
By BioPharma Dive staff -
Freeline, after cuts, agrees to take-private deal with Syncona
The planned acquisition adds to a string of bargain buyouts and reverse mergers in the gene therapy field.
By Ned Pagliarulo • Nov. 22, 2023 -
Gene editing
World’s first CRISPR medicine approved in UK for sickle cell, beta thalassemia
Clearance of Vertex Pharmaceuticals and CRISPR Therapeutics' Casgevy in the U.K. comes ahead of expected regulatory decisions in the U.S. and Europe.
By Ned Pagliarulo • Nov. 16, 2023 -
Ajinomoto spices up its biopharma business with gene therapy deal
Forge Biologics operates a contract manufacturing business and develops its own gene therapies, which appears to have attracted the Japan-based food and biotechnology company.
By Gwendolyn Wu • Nov. 13, 2023 -
Gene editing
First look at Verve study data offers base editing ‘proof of principle’
Early clinical trial results showed Verve’s therapy can substantially lower bad cholesterol. Still, investors sent the biotech’s shares down by 40% Monday.
By Ned Pagliarulo • Nov. 12, 2023 -
Deep Dive // Gene editing
What if a CRISPR cure isn’t such an easy choice?
A gene editing therapy developed by Vertex Pharmaceuticals and CRISPR Therapeutics can mute sickle cell disease’s most damaging symptoms. Yet treatment may not be as simple as its dramatic benefit makes it seem.
By Ned Pagliarulo , Shaun Lucas • Nov. 8, 2023 -
BioMarin to replace CEO Bienaimé with Genentech veteran
The rare disease drug developer is turning to former Genentech head Alexander Hardy as the launch of its Roctavian gene therapy stumbles.
By Kristin Jensen • Nov. 2, 2023 -
Vertex, CRISPR therapy for sickle cell passes FDA panel test
The high-profile meeting focused on the theoretical risks of CRISPR gene editing, as both the FDA and its advisory committee appeared convinced by the efficacy of the companies’ exa-cel treatment.
By Ned Pagliarulo • Oct. 31, 2023 -
A play-by-play of the FDA’s meeting on Vertex, CRISPR’s sickle cell therapy
Expert advisers dug deep on the risks of off-target gene editing and debated how best to set guidelines for the fast-growing field. Catch up on their full discussion here.
By Ned Pagliarulo , Jonathan Gardner , Gwendolyn Wu • Updated Oct. 31, 2023 -
Sarepta gene therapy for Duchenne misses main goal of key study
Results from the EMBARK study, which were meant to confirm the approval of Sarepta's Elevidys, sent the company's shares down more than 40% Tuesday.
By Ned Pagliarulo • Updated Oct. 31, 2023 -
Gene editing
FDA staff focus on ‘off-target’ risk in review of Vertex, CRISPR sickle cell therapy
Documents released ahead of a Tuesday advisory meeting show agency scientists to be generally convinced by the treatment’s benefit, a sign analysts read as positive.
By Ned Pagliarulo • Oct. 27, 2023 -
Gene editing
Vertex gearing up for launch as sickle cell therapy review advances
Executives at the biotech say they’re trying to get ahead of the payer and production challenges that will face their gene editing treatment exa-cel, which is now under FDA review.
By Jacob Bell • Oct. 26, 2023 -
Gene editing
Intellia cleared by FDA to start Phase 3 test of gene editing treatment
Developed with Regeneron, Intellia’s treatment is designed to inactivate a gene to treat an inherited disease called transthyretin amyloidosis.
By Gwendolyn Wu • Oct. 18, 2023 -
Deep Dive // Gene editing
A decade later, biotech’s CRISPR revolution is still going strong
Once the specialty of a few select drugmakers, CRISPR gene editing is now an essential technology for a growing group of biotechs, many led by former students of the field's pioneering scientists.
By Gwendolyn Wu , Shaun Lucas , Julia Himmel • Oct. 11, 2023 -
UniQure lays off 20% of staff, cuts research to ‘significantly’ lower costs
All told, the biotech said it will discontinue more than half of its research and technology projects, including a Parkinson’s disease treatment.
By Gwendolyn Wu • Oct. 5, 2023 -
Orchard sells to Kyowa Kirin in gene therapy buyout
Kyowa will pay nearly $400 million to acquire Orchard, which sells the gene therapy Libmeldy in Europe but has struggled to find paths to market for other experimental treatments.
By Ned Pagliarulo • Oct. 5, 2023 -
Regeneron, Intellia target neurological diseases in expanded gene editing deal
The longtime partners believe that, by combining their technologies, they can create “in vivo” genetic medicines for nervous system and muscular disorders.
By Jacob Bell • Oct. 3, 2023 -
Taysha drops lead gene therapy following FDA feedback
Additional studies proposed by the FDA would be too challenging, according to Taysha, which simultaneously announced that its partner Astellas has declined to pick up an option for the therapy, called TSHA-120.
By Kristin Jensen • Sept. 20, 2023 -
Orchard nears FDA decision on rare disease gene therapy
Three years after gaining European approval, Libmeldy is now under U.S. review with a deadline set for March.
By Jonathan Gardner • Sept. 18, 2023 -
UK biotech AlveoGene launches with plans for inhaled gene therapy
The company is working on a treatment of alpha-1 antitrypsin deficiency, which it claims it can deliver direct to lung cells via a nebulizer.
By Kristin Jensen • Sept. 14, 2023 -
Wave readies clinical testing for first RNA editing therapy
The biotech, one of several advancing RNA editing medicines, expects to obtain “proof-of-mechanism” data in patients with the inherited disorder alpha-1 antitrypsin deficiency by next year.
By Kristin Jensen • Updated Sept. 5, 2023 -
Beam begins US testing of first-of-its-kind ‘base editing’ cancer drug
The announcement marks progress for Beam after trial delays, and is the first time in the U.S. that a patient has received a base editing treatment.
By Ben Fidler • Sept. 5, 2023 -
Startup Cellares adds $255M as investors pour cash into cell therapy production
Bristol Myers Squibb is among those backing the company, which claims the manufacturing capacity at its New Jersey plant can surpass that of conventional CDMO facilities.
By Kristin Jensen • Aug. 24, 2023 -
Sponsored by MilliporeSigma
5 ways experience matters in gene therapy manufacturing
By partnering with an accomplished CTDMO, gene therapy innovators can benefit from guidance and support.
Aug. 21, 2023