Gene Therapy

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

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

Years later, a first-of-its-kind treatment shows the power, and limits, of gene therapy

Luxturna, approved four years ago as the first gene therapy for an inherited disease in the U.S., is improving sight and quality of life for several of the patients who received it.

By Shoshana Dubnow • Nov. 15, 2021

Eye-focused gene therapy startup gets $60M cash infusion from Sanofi

Shortly after postponing a $135 million IPO, Gyroscope has found another way to back a treatment for a disease that's become a top target of multiple drugmakers.  

By Jonathan Gardner • Nov. 8, 2021

Beam gets green light to begin first clinical test of base editing

Alongside the milestone, the gene editing biotech also announced John Maraganore, who will soon step down as Alnylam's CEO, will join its board.

By Ned Pagliarulo • Nov. 8, 2021

Blackstone continues biotech push with up to $250M cell therapy investment

The investment in CAR-T maker Autolus Therapeutics adds to a string of biotech deals the private equity firm has made since 2018.

By Ben Fidler • Nov. 8, 2021

Pfizer gene therapy research delayed by trial changes, safety questions

Data readouts for Pfizer's hemophilia gene therapies are now expected later than previously forecast, while the company also works through modifying a trial of its Duchenne treatment.

By Ned Pagliarulo • Nov. 3, 2021

FDA extends review of J&J, Legend's cell therapy for multiple myeloma

The three-month delay could give an advantage to Bristol Myers Squibb, which holds the first approval for a CAR-T therapy for the blood cancer.

By Jonathan Gardner , Ben Fidler • Nov. 2, 2021

Moderna, teaming with a startup, wades further into gene editing

The biotech has identified gene editing as a natural fit for the technology it uses to deliver its vaccines and drugs. A deal with Metagenomi, a startup backed by Bayer, will further those ambitions.

By Ben Fidler • Nov. 2, 2021

NIH, FDA spearhead broad partnership to speed gene therapy research

The two institutions will work with drugmakers and nonprofit groups on a wide-ranging initiative meant to make the complex treatments easier and less costly to produce. 

By Kristin Jensen • Oct. 28, 2021

Vertex aligns with another CRISPR gene editing startup

A new alliance with Mammoth Biosciences marks the latest evidence of Vertex's interest in gene editing, which has already been the focus of multiple deals with emerging biotechs. 

By Ben Fidler • Oct. 26, 2021

Bluebird, winding down in Europe, withdraws another rare disease gene therapy

The biotech won EU approval for two gene therapies, Zynteglo and Syksona, both of which it will pull from market after difficulties negotiating reimbursement.

By Ned Pagliarulo • Oct. 21, 2021

Boehringer Ingelheim takes next step in development of cystic fibrosis gene therapy

Drugs from Vertex can effectively treat the lung disease in as much as 90% of patients. Gene therapy could be a solution for the remaining 10%, but the path forward is challenging.

By Shoshana Dubnow • Oct. 19, 2021

Takeda takes aim at a biotech's gene therapy work

For an upfront payment of $45 million, Takeda gains access to as many as eight programs from Poseida Therapeutics and a potential method of delivering gene therapies without the help of viruses.

By Jacob Bell • Oct. 12, 2021

Intergalactic Therapeutics launches with $75M to build a new type of gene therapy

Founded by former Biogen executive Michael Ehlers and backed by Apple Tree Partners, Intergalactic aims to create non-viral gene therapies delivered via an advanced form of electroporation.

By Ned Pagliarulo • Oct. 7, 2021

Gene therapy developer gets chance at a comeback with Pfizer deal

Voyager Therapeutics recently scrapped much of its pipeline and replaced top executives. Now, a Pfizer deal on new technology gives investors hope.

By Jonathan Gardner • Oct. 6, 2021

Sarepta embarks on late-stage clinical trial of Duchenne gene therapy

Initiation of the Phase 3 trial is an important milestone for the biotech after earlier setbacks, as well as for patients with the inherited muscle disease.

By Shoshana Dubnow • Oct. 4, 2021

Amicus, looking for a boost, sells its gene therapy work to a SPAC

The value of the biotech's growing gene therapy portfolio has been "unrecognized" by investors, said CEO John Crowley, leading to a sudden shift for an initiative that began just three years ago.

By Ben Fidler • Sept. 29, 2021

Worrisome side effects lead Pfizer to narrow Duchenne gene therapy trial

Outside trial monitors have attributed three cases of severe muscle weakness to Pfizer's treatment, causing the company to change the design of its closely watched Phase 3 study.

By Ben Fidler • Sept. 28, 2021

FDA lifts hold on GeneTx, Ultragenyx study of Angelman therapy

The hold, which was put in place during the fourth quarter last year, came after five patients in an early trial experienced muscle weakness following treatment.

By Shoshana Dubnow • Sept. 27, 2021

Novartis pushes further into gene therapy for the eye with deal for Swiss startup

The large drugmaker has acquired Arctos Medical and its preclinical research into optogenetics, an area Novartis already showed interest in with its buyout of Vedere Bio last year.

By Ned Pagliarulo • Updated Sept. 23, 2021

Fourth trial volunteer dies in Astellas gene therapy study

The study, which was suspended following three deaths last year, had been restarted in February after Astellas lowered the treatment dose used. 

By Ned Pagliarulo • Sept. 14, 2021

AbbVie bets big on a gene therapy for eye diseases

The company has agreed to pay $370 million, and possibly north of $1 billion more, to gain access to a Regenxbio treatment targeting wet AMD and other eye conditions.

By Jacob Bell • Sept. 13, 2021

Cell and gene therapies' evolving temperature-controlled requirements call for specialized logistics solutions

The CGT industry is expected to grow 30% between 2019 and 2025, and the transition from clinical trials to commercial-scale production is now seen as inevitable.

By Leandro Moreira, Yourway • Sept. 13, 2021

Lilly joins RNA editing race with ProQR deal

An agreement with the Dutch biotech, which could be worth as much as $1.3 billion, expands Lilly's work in genetic medicine research.

By Kristin Jensen • Sept. 9, 2021