Gene Therapy: Page 3
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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 -
Trendline
Gene 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 -
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 -
Bruker to buy PhenomeX and its cell research tools for $108M
The company said PhenomeX’s technology will help its customers speed development and manufacturing of cell and gene therapies.
By Susan Kelly • Aug. 18, 2023 -
FDA won’t hold advisory meeting for Bluebird’s sickle cell gene therapy
The decision stands in contrast to the agency’s plans for a would-be rival gene editing treatment from Vertex Pharmaceuticals and CRISPR Therapeutics that is also under a regulatory review.
By Kristin Jensen • Aug. 16, 2023 -
Precision sells lymphoma drug to Imugene in retreat from cell therapy research
The struggling biotech also plans to offload other cell therapy assets, including a multiple myeloma therapy, and focus exclusively on gene editing medicines.
By Ben Fidler • Aug. 16, 2023 -
HIV cell therapy startup Addimmune heads to Wall Street via blank-check merger
The deal will help fund a cell-based treatment that completed Phase 1 testing last year and is designed to provide a “functional cure” for patients with HIV infections.
By Ben Fidler • Aug. 10, 2023 -
Regeneron ups genetic medicine investment with Decibel buyout
Third Rock-founded Decibel, which has shed much of its value since a 2021 IPO, expected to run out of money within a year.
By Jonathan Gardner • Aug. 9, 2023 -
Sarepta offers early look at closely watched gene therapy launch
One patient received its Duchenne gene therapy, Elevidys, this week and a handful more could follow shortly, surprising some analysts and raising near-term sales expectations.
By Ben Fidler • Aug. 3, 2023 -
A once buzzy gene therapy startup lays off staff and shuts down
Intergalactic, a developer of non-viral gene therapies built by ex-Biogen executive Michael Ehlers, is folding less than two years after its launch.
By Kristin Jensen • Aug. 2, 2023 -
Vertex taking ‘flexible’ approach to payment talks for experimental CRISPR therapy
The company is discussing various reimbursement models for its sickle cell treatment with government and commercial insurers ahead of an FDA approval decision later this year.
By Ned Pagliarulo • Aug. 2, 2023 -
AstraZeneca buys Pfizer’s early gene therapy work for up to $1B
The British pharma is deepening its investment in genetic medicine through a deal for a group of Pfizer’s gene therapy delivery tools, while the New York giant cuts back.
By Ben Fidler • July 28, 2023 -
Kincell spins out of cell therapy biotech, joining wave of manufacturing startups
The company is the latest to emerge with plans to make production of cell-based medicines easier for researchers and biotechs.
By Ben Fidler • July 26, 2023 -
Gene therapy biotech Kriya adds $150M in fresh funds
Led by Vivek Ramaswamy's brother, Kriya has now raised over $600 million to fund gene therapy research spanning ophthalmology, neurology and metabolic disease.
By Kristin Jensen • July 26, 2023 -
Editas names Biogen veteran as top scientist in latest executive change
Linda Burkly’s appointment follows the exit of CSO Mark Shearman, whose departure Editas announced alongside layoffs earlier this year.
By Gwendolyn Wu • July 24, 2023 -
Sponsored by Center for Breakthrough Medicines
Cell and gene therapy is coming of age
Outsourcing in the cell and gene therapy industry is booming and no longer considered just a “trend.”
July 24, 2023 -
A cancer biotech’s collapse gives Neurogene a path to Wall Street
The startup, a developer of gene therapies for brain diseases like Rett syndrome, will bypass an IPO by merging with struggling drugmaker Neoleukin.
By Delilah Alvarado • July 18, 2023