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

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

Explore the 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

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

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

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

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