FDA approves Krystal gene therapy for rare wound disorder

Cleared to treat dystrophic epidermolysis bullosa, Vyjuvek is the sixth gene therapy for an inherited disease to gain clearance in the U.S.

By Delilah Alvarado • May 22, 2023

Novartis buys rare disease gene therapy from Avrobio

The pharma will pay nearly $90 million to acquire Avrobio’s treatment for cystinosis, an inherited condition caused by the toxic buildup of an amino acid.

By Ned Pagliarulo • May 22, 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

Mustang pares research, sells manufacturing plant to save money

The biotech joins a score of other cell and gene therapy developers that, on the heels of a historic market downturn, have cut costs.

By Jacob Bell • May 18, 2023

Siren emerges from stealth with plans to marry gene therapy to cancer immunotherapy

Spun out of the lab of former UCSF researcher Nicole Paulk, the biotech is targeting brain and eye tumors with a new kind of treatment it has dubbed “immuno-gene therapy.”

By Ned Pagliarulo • May 17, 2023

Gene therapy consortium targets eight rare diseases for clinical trials

Backed by nearly $100 million, the public-private consortium aims to create a standard development roadmap for gene therapies using AAV viral vectors.

By Christopher Newman • Updated May 16, 2023

UniQure sells royalty rights to hemophilia gene therapy

The Dutch biotech sold a portion of royalties owed by CSL Behring on sales of Hemgenix, adding to a cash pile it has built since out-licensing the treatment.

By Christopher Newman • May 15, 2023

FDA panel narrowly backs approval of Sarepta’s Duchenne gene therapy

Shares in Sarepta rose by 25% Monday following a close advisory committee vote supporting accelerated approval of the biotech’s treatment, despite uncertainty around its benefit.

By Ned Pagliarulo , Ben Fidler • Updated May 12, 2023

FDA staff skeptical of Sarepta’s Duchenne gene therapy, documents show

Sarepta has not provided “unambiguous evidence” its treatment can help patients, agency scientists wrote in documents released ahead of a crucial Friday advisory committee meeting.

By Ben Fidler • Updated May 10, 2023

‘The luckiest of the unlucky’: A Duchenne gene therapy brings hope to families — and tests the FDA

The FDA in June approved a treatment that patient advocates and doctors believe to be a breakthrough for a deadly disease, but hasn’t yet proven that it works. 

By Ben Fidler , Shaun Lucas • May 7, 2023

After delay, Bluebird submits sickle cell gene therapy for FDA approval

Submission of the application comes weeks after rivals Vertex and CRISPR filed their gene editing medicine for the disease with the agency.

By Christopher Newman • April 24, 2023

French biotech pulls EMA application for eye disease gene therapy

With cash running low and a search for a buyer underway, GenSight wants advice from regulators on the type of data that would support an approval.

By Jonathan Gardner • April 21, 2023

Function Oncology raises $28M to use CRISPR to tailor cancer diagnosis and treatment

The startup says its genomics platform can improve on conventional sequencing by uncovering drug target vulnerabilities directly in tumor samples.

By Nick Paul Taylor • April 13, 2023

Century Therapeutics changes leadership as CEO steps down

The resignation of top executive Lalo Flores comes three months after a restructuring, marking the latest change of direction for the once high-flying cell therapy startup.  

By Delilah Alvarado • April 12, 2023

A new biotech wants to ease a bottleneck in cell and gene therapy production

Founded by University of Pennsylvania researchers, VintaBio will manufacture the viral vectors that are essential in cell and gene therapy production, but which have been in shorter supply as more companies entered the space.

By Jacob Bell • April 11, 2023

GSK outlines deal to send cell therapies back to Adaptimmune

The pharma, which recently retreated from cell therapy research, will pay Adaptimmune about $37 million as part of an agreement to return rights to two cancer treatments.

By Ben Fidler • April 11, 2023

Ginkgo grows its gene therapy offerings with StrideBio deal

The deal hands Ginkgo technology for discovering and engineering capsids — the outer shell that protects the helpful genetic material in gene therapies.

By Jacob Bell • April 5, 2023

Freeline cuts more jobs and halts Fabry gene therapy work

The U.K. biotech will lay off 30% of its workforce and focus on an experimental Gaucher disease treatment, five months after an initial restructuring that cut a hemophilia gene therapy.

By Christopher Newman • April 4, 2023

Vertex, CRISPR finish US filing for gene editing drug approval

The approval application is the first in the U.S. for a CRISPR-based medicine and puts the partners ahead of a rival therapy from Bluebird bio. 

By Christopher Newman • April 3, 2023

Changing cell & gene therapy landscape and implications on clinical trials

Cell & gene therapy clinical research may help treat diseases and change how we approach healthcare.

By Elizabeth Dugan, Director, Business Development • April 3, 2023

Gene therapy startup Vedere to close two years after launch

The startup, a successor to a biotech that Novartis bought in 2020, will wind down after preclinical experiments didn’t meet the bar set by its leaders.

By Ben Fidler • Updated April 2, 2023

Regeneron bets on ‘Treg’ cell therapy with Sonoma deal

The biotech will work with Sonoma, a well-funded startup run by prominent immunologist Jeffrey Bluestone, to develop cell-based medicines for ulcerative colitis and Crohn’s disease.

By Ben Fidler • March 28, 2023

Vertex pays CRISPR to use its gene editing tech for diabetes drugs

Vertex will license CRISPR technology to develop insulin-producing islet cells that are more resistant to immune rejection, adding to other efforts directed at Type 1 diabetes.

By Christopher Newman • March 27, 2023

Moderna inks second genetic medicine deal in a month

The biotech plans to use Generation Bio’s nonviral delivery technology to get nucleic acid-based therapies to immune cells and into the liver.

By Christopher Newman • March 23, 2023

Gilead’s Yescarta extends survival in lymphoma study

The results, the first for a CAR-T therapy, are further evidence supporting the use of the complex therapies earlier in a patient’s disease course. 

By Ben Fidler • March 21, 2023

Addressing manufacturing challenges in gene therapy development

As new gene therapies are developed to treat a growing number of indications, patients may soon have access to novel treatment options and potential cures.

By Kate Silver • March 20, 2023