Gene Therapy: Page 2

5 questions facing gene therapy in 2021

Gene therapy developers will need to address a series of setbacks that have shaded optimism in the sector's fast growth. Dealmaking, however, appears to be continuing apace. 

By Ned Pagliarulo • Jan. 08, 2021

Sarepta gene therapy misses goal in key muscular dystrophy study

Company executives, however, blamed "improbably bad luck" for study enrollment that resulted in more older patients with milder disease receiving placebo, and pledged to press on.

By Jonathan Gardner • Jan. 07, 2021

Explore the Trendline➔

Trendline

Gene Therapy

Rapid scientific advances have put the gene therapy field at the forefront of biomedical research. But, as recent setbacks have shown, reserachers and drugmakers still face major challenges. 

By BioPharma Dive staff

Pfizer beats Sarepta to start of first late-stage Duchenne gene therapy trial

Sarepta, though, may try to seek accelerated approval using Phase 2 data that's due imminently, along with results from another, smaller trial.

By Jonathan Gardner • Jan. 07, 2021

Biogen pushes further into eye gene therapy with new deal

Partnering with German biotech ViGeneron bolsters Biogen's pipeline of drugs for eye diseases, which includes assets acquired through its buyout of Nightstar Therapeutics.

By Kristin Jensen • Jan. 06, 2021

Cancer case puts UniQure's hemophilia gene therapy on hold, raising alarms

The FDA halted the AMT-061 program, which could be the first approved gene therapy for hemophilia B, because a patient who received the treatment appears to have developed liver cancer.

By Jacob Bell • Dec. 21, 2020

Lilly pays $880M for Prevail, pushing further into genetic medicine

The acquisition marks a significant bet by Lilly on gene therapy's potential to address tough-to-treat neurological disorders like Parkinson's disease.

By Jonathan Gardner • Dec. 15, 2020

UniQure offers a closer look at the leading hemophilia B gene therapy

Doctors on Tuesday got a more detailed idea of how the therapy works and who might be eligible to take it, as late-stage results were presented at ASH.

By Jacob Bell • Dec. 08, 2020

Bayer strikes a deal with Atara, wading deeper into cell therapy

A pact for two Atara programs continues the German pharma's efforts to use dealmaking to build a gene and cell therapy division.

By Ben Fidler • Dec. 07, 2020

4 storylines to track at the year's biggest meeting on blood diseases

A closely watched gene editing therapy, a slate of new multiple myeloma drugs and much more are set to highlight the American Society of Hematology conference this weekend.

By Ben Fidler , Ned Pagliarulo , Jacob Bell • Dec. 03, 2020

J&J pushes further into eye gene therapy

The pharma licensed a treatment for a severe type of age-related vision loss, building on past deals that gave it a foothold in gene therapy.

By Jacob Bell • Dec. 02, 2020

Regeneron, UPenn to use gene therapy tools to deliver a COVID-19 antibody drug

Working with University of Pennsylvania researcher Jim Wilson, Regeneron aims to develop a nasal spray version of the coronavirus treatment recently cleared by the Food and Drug Administration. 

By Kristin Jensen • Dec. 01, 2020

Lilly dives into gene editing

Through a deal with Precision Biosciences, the pharma aims to develop therapies for genetic disorders, including Duchenne muscular dystrophy.

By Jacob Bell • Nov. 20, 2020

UniQure's gene therapy for hemophilia B meets goal in key study

The results are first to emerge from a late-stage test of a hemophilia B gene therapy, and show UniQure's treatment can restore clotting protein levels to mild or even near normal levels.

By Ned Pagliarulo • Nov. 19, 2020

UCB strikes 2 gene therapy deals to build its pipeline

With patent expirations nearing for a top-selling drug, the Belgian pharma will buy one company and partner with another to add several early-stage projects in neurological diseases.

By Jonathan Gardner • Nov. 12, 2020

Intellia, beginning treatment in CRISPR study, secures Gates Foundation backing

The biotech won a grant from the foundation two days after dosing the first patient in a study of an in vivo CRISPR medicine, a milestone for the company and the technology. 

By Kristin Jensen • Nov. 11, 2020

Pfizer invests in Homology, adding to gene therapy ambitions

Along with an equity stake, Pfizer will get right of first refusal on any deal involving Homology's most advanced therapies, which the biotech is developing for a rare metabolic disorder.

By Kristin Jensen • Nov. 10, 2020

FDA gene therapy holdups suggest closer scrutiny by agency

Requests for more data on manufacturing, clinical holds and a surprise rejection have led to questions of a tougher standard at the agency. Officials, however, point to a large and fast-advancing pipeline.

By Jonathan Gardner • Nov. 05, 2020

Gene therapy for Duchenne muscular dystrophy: nearing final tests

Sarepta and Pfizer are advancing their experimental treatments into late-stage trials, while others are accelerating research in what's become one of gene therapy's most active fields.

By Ben Fidler • Nov. 05, 2020

Bluebird faces delay for sickle cell gene therapy as rivals inch closer

The setback for Bluebird's LentiGlobin comes amid a string of manufacturing-related delays for gene therapy developers.

By Kristin Jensen • Nov. 05, 2020

Axovant shares sink on manufacturing delay for Parkinson's gene therapy

A longer-than-expected timeline for developing a new manufacturing process means a key study for Axovant's top gene therapy likely won't begin until 2022.

By Ned Pagliarulo • Oct. 30, 2020

Novartis eyes its next move in gene therapy

Acquiring Atlas Venture-backed startup Vedere Bio gives Novartis a technology it believes will be valuable for developing treatments for many causes of vision loss and blindness.

By Jacob Bell • Oct. 29, 2020

Bayer to buy AskBio for $2B in bid to become a gene therapy player

Founded by gene therapy pioneer Jude Samulski, AskBio boasts a pipeline of experimental gene therapies as well as its own manufacturing capabilities.

By Ben Fidler • UPDATED: Oct. 26, 2020 at 11:55 a.m.

Ultragenyx expands into Duchenne gene therapy with Solid Bio deal

The rare disease drug developer is betting it can create a better DMD treatment by combining Solid's research with its manufacturing and gene delivery technology.

By Ned Pagliarulo • Oct. 23, 2020

Sarepta, continuing its gene therapy push, helps launch a startup

The biotech has joined a group of investors to form a new gene therapy developer led by one of its longtime executives and backed with $107 million. 

By Jacob Bell • Oct. 22, 2020

Intellia cleared to start key CRISPR drug study in UK

The trial would be the first test of a systemically administered gene editing treatment that uses the Nobel Prize-winning technology to alter DNA.

By Ned Pagliarulo • Oct. 19, 2020