Gene Therapy: Page 2

Amicus' SPAC deal unravels, leading to layoffs and cost cuts

The biotech is calling off plans to spin out its gene therapy business via a merger with a blank check company, citing unfavorable market conditions and a "challenging environment."

By Kristin Jensen • Feb. 24, 2022

Allogene, slipping in 'off-the-shelf' CAR-T race, prepares for key trials

Allogene, once the clear leader in development of a more convenient option to personalized cancer cell therapies, now is competing with CRISPR Therapeutics and other fast-emerging rivals.

By Ben Fidler • Feb. 24, 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

Spark CEO Marrazzo, who led company to historic gene therapy approval, to step down

Jeffrey Marrazzo has led Spark since its founding in 2013, through the historic FDA approval of Luxturna and the company's $4.3 billion buyout by Roche.

By Ned Pagliarulo • Feb. 23, 2022

BioMarin says gene therapy hold will endure as FDA questions cancer risk

According to BioMarin, the regulator has requested additional data to assess the "theoretical" cancer risk to the treatment, information that will take several quarters to collect. 

By Ned Pagliarulo • Feb. 18, 2022

Intellia follows CRISPR peers with deal to explore 'natural killer' cell therapy

By partnering with ONK Therapeutics, Intellia joins CRISPR Therapeutics and Editas Medicine in striking deals that marry gene editing with a fast-emerging form of cancer immunotherapy.

By Ben Fidler • Feb. 15, 2022

Private equity powers digital healthcare revolution

Read how private equity investment helps scale the push towards healthcare digitization.  

By First Republic Bank • Feb. 7, 2022

In England, Orchard secures rare agreement on gene therapy coverage

Gene therapy developers have struggled to secure reimbursement in Europe, making Orchard's deal with the National Health Service in England and Wales a notable accomplishment.

By Ned Pagliarulo • Feb. 4, 2022

Sarepta reveals gene editing alliance with biotech exploring alternative delivery methods

The collaboration with GenEdit, a South San Francisco startup developing new ways of delivering gene editing tools into the body, dates back to December 2020, but has recently begun to advance.

By Ned Pagliarulo • Feb. 1, 2022

Bluebird faces longer wait for FDA verdict on experimental gene therapies

The FDA has extended by three months its review of Bluebird's treatments for beta thalassemia and a rare brain disorder, the latest delay in the company's lengthy road to market in the U.S.

By Ned Pagliarulo • Jan. 18, 2022

5 questions facing gene therapy in 2022

As record levels of money pour into gene therapy research, biotechs are under increased pressure to address issues related to safety, delivery and overlapping pipelines. 

By Ned Pagliarulo • Jan. 13, 2022

Sarepta, with new Duchenne results, to broach 'possibility' of speedy gene therapy approval

The biotech will take new Phase 2 data to U.S. regulators, CEO Doug Ingram said Monday, though it still expects to need data from an ongoing late-stage trial to win approval. 

By Ben Fidler • Jan. 10, 2022

Allogene cleared by FDA to resume 'off the shelf' cancer cell therapy trials

A "chromosomal abnormality" that led regulators to halt Allogene's trials was judged to be unrelated to its technology, a finding with important implications for the field of donor-derived cell therapies.

By Ben Fidler • Jan. 10, 2022

BioMarin plans return to FDA with updated data on hemophilia gene therapy

Two-year results from a Phase 3 study of Roctavian show treatment prevented bleeding, potentially giving BioMarin the data it needs to resubmit an application for approval. 

By Ned Pagliarulo • Updated Jan. 10, 2022

Pfizer expands into gene editing with Beam research deal

Pfizer's work with mRNA vaccines led it to explore other applications of the technology, resulting in a multiyear partnership with the high-profile biotech Beam Therapeutics on gene editing treatments for rare diseases.

By Ned Pagliarulo • Jan. 10, 2022

Sanofi cuts ties with Sangamo, sharpening focus on 'off-the-shelf' cell therapy

The French pharma cited a "change in strategic direction" for ending a long-running pact focused on genetic medicines for blood diseases, led by a sickle cell treatment in early testing.

By Ben Fidler • Jan. 6, 2022

Novartis ups investment in gene therapy for the eye with $800M buyout

The deal for Gyroscope Therapeutics, worth up to $1.5 billion, gives the Swiss pharma access to a treatment for a type of age-related blindness as well as new means of delivering drugs into the eye.

By Jonathan Gardner • Dec. 22, 2021

Duchenne patient dies in Pfizer gene therapy study

The tragic news follows changes Pfizer recently made to the design of another study testing the therapy due to side effects seen in some participants. 

By Jonathan Gardner • Dec. 21, 2021

Bluebird hit with another hold for sickle cell gene therapy

Study enrollment and treatment of sickle cell patients under 18 will be paused for safety reasons, another hurdle in a series of setbacks for the company.

By Ned Pagliarulo • Dec. 20, 2021

Spark expands in Philadelphia, with new facility meant to be Roche's gene therapy hub

The Luxturna developer, now owned by Roche, will invest $575 million to build a huge research and development center on Drexel University's campus.

By Ned Pagliarulo • Dec. 17, 2021

Pfizer opens gene therapy plant in $800M North Carolina expansion

The new facility cost nearly $70 million to build, and is part of a major push by the pharma giant to become a leader in genetic medicine manufacturing.

By Kristin Jensen • Dec. 16, 2021

Rebounding from setbacks, Sanofi reveals promising data for hemophilia drug

Fitusiran, which Sanofi licensed from Alnylam, could finally get to market after trial delays and safety concerns slowed its progress. But gene therapies and other new medicines might provide competition.

By Jonathan Gardner • Dec. 14, 2021

Fresh data show UniQure's hemophilia gene therapy appears to hold up

The amount of blood clotting protein found in patients who received the medicine was nearly the same at six months and 18 months post-treatment, according to results disclosed Thursday.

By Jacob Bell • Dec. 9, 2021

A high-profile gene therapy biotech takes aim at Huntington's

Passage Bio and the University of Pennsylvania's Gene Therapy Program said they have begun preclinical research aimed at simultaneously developing multiple approaches to treating Huntington's.

By Jacob Bell • Dec. 8, 2021

Heart attacks struck Sek Kathiresan's family. He's devoted his life to stopping them.

After several family members had early heart attacks, Kathiresan vowed to understand why they happen. His research journey has changed medical practice and led to a new biotech startup, Verve Therapeutics, that seeks to prevent them.

By Ben Fidler • Nov. 29, 2021

Bluebird, after delays, gets speedy FDA review for beta thalassemia gene therapy

The regulator will decide whether to approve Bluebird's treatment by next May, but its evaluation is beginning more than a year later than the biotech hoped.

By Ned Pagliarulo • Nov. 22, 2021