Gene Therapy: Page 2

Editas names new chief medical officer in latest executive change-up

Baisong Mei, a veteran of Sanofi and Biogen, comes to Editas half a year after the gene editing company fired his predecessor and one month after new CEO Gilmore O'Neill started on the job.

By Christopher Newman • Updated July 18, 2022

Verve starts first human test of gene editing treatment for heart disease

A patient in New Zealand became the first person to receive the biotech’s medicine, which uses base editing to turn off a specific gene in the liver and thereby lower cholesterol.

By Ned Pagliarulo • July 12, 2022

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

C. difficile infection: Current treatment options and challenges

Clostridioides difficile infection is a highly contagious disease that affects 500,000 people in the U.S. and results in nearly 30,000 deaths per year.

By Dr. Teena Chopra • July 11, 2022

Adverum cuts jobs, restructures to give eye gene therapy another shot

A year after reporting side effects “not seen before in ocular gene therapy” in a clinical trial, the biotech is eliminating 38% of its workforce to save cash while it runs a new study. 

By Ben Fidler • July 7, 2022

J&J-backed gene therapy for the eye clears early study test

Positive results for the treatment, which was developed by U.K. biotech MeiraGTx, could rebuild expectations after another gene therapy from Biogen failed in the retinal disease.

By Ned Pagliarulo • June 28, 2022

Another Astellas gene therapy trial paused by FDA after side effect report

The regulator suspended testing of Astellas’ Pompe disease treatment after one patient experienced peripheral nerve damage. Both of the pharma’s clinical-stage gene therapies are now on hold.

By Ben Fidler • June 27, 2022

C. difficile infection: A close-up on an urgent public health threat

As hospitals have dealt with surging admissions and extended stays with COVID-19, staff and patients alike have still had to contend with another potentially deadly infection from a bacterium called Clostridioides difficile.

By Dr. Dennis Deruelle • June 27, 2022

BioMarin's hemophilia gene therapy recommended for approval in Europe

The decision brings what could be the first approved hemophilia gene therapy, Roctavian, closer to market, after a series of regulatory setbacks that have delayed its arrival. 

By Jacob Bell • June 24, 2022

UniQure buoyed by early data for Huntington's gene therapy

After one year, researchers detected important protein changes in patients who received a low dose of the experimental treatment. Further testing and functional data are needed to assess its potential, however.

By Jonathan Gardner • June 23, 2022

Novartis hunting for sickle cell cure with Precision deal

The pharma will pay $75 million to access Precision’s gene editing technology, adding another program to the lengthening list of drug development efforts targeting the inherited blood condition.

By Ned Pagliarulo • June 22, 2022

A biotech startup launches with $38M to develop a cystic fibrosis gene therapy

Carbon Biosciences believes its technology, which is based on parvoviruses, could allow for larger gene delivery and repeat dosing.

By Delilah Alvarado • June 21, 2022

Breaking down the role of the gut microbiome

When it comes to the human body, we are never truly alone. In fact, we share our bodies with a diverse and complex colony of microorganisms, called the microbiome.

By Dr. Carl Crawford (GI) • June 21, 2022

Vertex, CRISPR strengthen case for pioneering gene-editing treatment

Positive data from 75 patients with either sickle cell disease or beta thalassemia keep the companies’ therapy on track to become the first CRISPR-based treatment submitted to drug regulators, possibly by the end of the year.

By Jacob Bell • June 11, 2022

FDA advisers offer unanimous support to second Bluebird gene therapy

By a 13-0 vote, the panel agreed the benefits of Bluebird’s beta thalassemia treatment outweigh its risks, one day after reaching a similar conclusion for another of the company’s drugs.

By Ned Pagliarulo • Updated June 10, 2022

Latest Caribou data add to 'off-the-shelf' cell therapy's durability questions

Three of the six lymphoma patients who received Caribou’s gene editing treatment have relapsed in the latest sign that so-called allogeneic drugs could have trouble matching their CAR-T counterparts. 

By Ben Fidler • June 10, 2022

FDA panel backs Bluebird gene therapy despite safety risks

In a major win for Bluebird, advisers to the agency voted 15-0 in support of its rare disease treatment eli-cel, downplaying concerns raised by FDA staff.

By Ned Pagliarulo • Updated June 9, 2022

Bluebird's future in balance as FDA weighs gene therapy approvals

Once among biotech’s most valuable companies, Bluebird is running out of cash. Its fate could rest on the FDA’s review of two rare disease treatments, which are being discussed at a two-day meeting that continues Friday. 

By Ned Pagliarulo • June 9, 2022

Astellas, despite recent setbacks, opens $100M gene therapy plant

The opening of the plant, which could create more than 200 new jobs, comes on the heels of several significant issues with the company’s most advanced gene therapy program.

By Jacob Bell • June 8, 2022

Immatics and Editas join up to bring CRISPR to 'gamma delta' cell therapy

The two biotechs will use gene editing to make prospective treatments more potent, adding to a recent flurry of activity involving the fast-emerging form of cellular immunotherapy.

By Ben Fidler • June 7, 2022

BioMarin delays planned FDA filing for hemophilia gene therapy

The California biotech now expects to resubmit its approval application to the FDA by the end of September, the latest regulatory setback for its closely watched treatment. 

By Ned Pagliarulo • May 31, 2022

FDA starts clock on review of CSL, UniQure gene therapy

The regulator agreed to assess the companies' data under priority review, setting up a decision on approval of the hemophilia B treatment in six months.

By Ned Pagliarulo • May 25, 2022

PTC's first gene therapy gains European regulator's backing

The brain-infused treatment for a rare neurological disease would be one of only three gene therapies commercially available in the EU. PTC plans to ask for FDA approval later this year.

By Jonathan Gardner • May 20, 2022

Scientists home in on cause of Duchenne gene therapy side effect

An unusual collaboration among gene therapy developers suggests certain mutations could be behind "peculiar" side effects experienced by several patients treated in clinical trials.

By Jonathan Gardner • Updated May 18, 2022

Ultragenyx strikes deal with struggling Abeona for rare disease gene therapy

The treatment, which is designed to treat Sanfilippo syndrome, could become Ultragenyx's first gene therapy to reach market in the U.S., according to the biotech's CEO.

By Ned Pagliarulo • May 17, 2022

Pfizer cleared to restart hemophilia gene therapy trial

While a voluntary pause in dosing new patients will remain in place, the FDA's decision puts the study back on track to deliver data in 2023.

By Jonathan Gardner • May 3, 2022