Sickle cell pipeline narrows as gene therapy developers rethink research plans

Graphite Bio and Sangamo are stopping work on their respective sickle cell gene therapies, while Intellia revealed partner Novartis ended development of its genetic treatment for the blood disease.

By Ned Pagliarulo • Feb. 23, 2023

Medicaid, with planned payment pilot, girds for influx of pricey gene therapies

The proposed model could help state Medicaid agencies explore different kinds of outcomes-based payment schemes, but may come too late to prepare for the first sickle cell gene therapy.

By Ned Pagliarulo • Feb. 21, 2023

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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

Talaris restructures, ending effort to develop a cell therapy for kidney transplants

Following a patient death and difficulty enrolling study participants, the company is laying off a third of its staff and ending a pair of studies for a two-decade-old program once owned by Novartis.

By Ben Fidler • Feb. 16, 2023

With Aera, CRISPR pioneer Feng Zhang goes after gene therapy’s delivery problem

The company’s platform technology is built on research from Zhang’s lab at the Broad Institute, which focuses on proteins that could be used to package and deliver nucleic acids.

By Gwendolyn Wu • Feb. 16, 2023

Astellas switches CEOs amid plans to pursue ‘aggressive’ growth

Naoki Okamura, currently Astellas’ chief strategy officer, will replace current CEO Kenji Yasukawa as the company continues a strategic reboot that’s seen it invest more heavily in gene therapy.

By Christopher Newman • Feb. 6, 2023

Roche writes off $3B on lower sales forecasts for gene therapies, cancer drugs

Among the drugs Roche recorded impairment charges on are four gene therapies acquired in the company's 2019 deal for Spark Therapeutics. 

By Jonathan Gardner • Feb. 3, 2023

Magenta to explore sale, merger after study setback

The biotech recently stopped a trial of its experimental conditioning regimen over safety concerns. Now it’s halting further development work as it undertakes a strategic review.

By Gwendolyn Wu • Feb. 3, 2023

Head of FDA gene therapy office set to retire in March

Wilson Bryan, a key decision maker in the agency’s oversight of genetic medicines, is expected to leave next month amid an organizational revamp of his office, the FDA confirmed.

By Kristin Jensen • Feb. 1, 2023

UniQure nabs another gene therapy for ALS

For $10 million upfront, UniQure has licensed rights to its second experimental treatment for the condition, a medicine from startup Apic Bio that should enter human testing later this year.

By Jacob Bell • Jan. 31, 2023

J&J, Legend’s cancer cell therapy has early success in key clinical trial

The trial is an important test of Carvykti’s potential for earlier use in multiple myeloma and found treatment helped extend progression-free survival over standard regimens.

By Jacob Bell • Jan. 27, 2023

Magenta halts study of targeted conditioning drug after participant’s death

The biotech said one patient died after receiving an experimental antibody drug designed to deplete certain blood and bone marrow cells before a transplant.

By Christopher Newman • Updated Jan. 26, 2023

Sales of J&J, Legend cell therapy plateau amid production challenges

Carvykti, which was approved a year ago for hard-to-treat multiple myeloma, generated $55 million in fourth-quarter sales, on par with the previous quarter.

By Jacob Bell • Jan. 25, 2023

FDA lifts hold on Astellas gene therapy for Pompe disease

The regulator’s decision ends a seven-month study pause that followed a report of one participant experiencing mild symptoms of peripheral neuropathy.

By Christopher Newman • Jan. 20, 2023

Al Sandrock on his short retirement and taking on a biotech turnaround project

In a conversation at the J.P. Morgan Healthcare conference, the longtime Biogen executive discussed his hesitance to jump back into an executive role and why an opportunity to run Voyager Therapeutics drew him in.

By Ben Fidler • Jan. 17, 2023

EMA weighs new safety guidance for Novartis’ gene therapy Zolgensma

The discussions come months after two patients died from acute liver injury following treatment with the spinal muscular atrophy therapy.

By Delilah Alvarado • Jan. 13, 2023

5 questions facing gene therapy in 2023

Buoyed by recent approvals, the field faces a pivotal year that’s likely to bring new treatments as well as more challenges.

By Ned Pagliarulo • Jan. 12, 2023

Pfizer pares back early-stage research in rare disease, cancer

The drugmaker said it is considering licensing out some assets to shorten development timelines, and will stop work on early viral-based gene therapies. 

By Jonathan Gardner • Jan. 6, 2023

Pfizer says hemophilia B gene therapy controlled bleeding in key study

The Phase 3 results showed the one-time treatment outperformed standard factor replacement in adults with the bleeding disorder, but suggest it may be less potent than CSL and UniQure’s rival therapy. 

By Delilah Alvarado • Dec. 29, 2022

Ferring wins FDA approval for bladder cancer gene therapy

The OK caps a long development journey for the treatment, which was previously turned back by the agency. Ferring doesn’t expect it to be available until the second half of next year, however.

By Delilah Alvarado • Dec. 19, 2022

FDA lifts hold on Bluebird’s sickle cell gene therapy

Removal of the partial study suspension, which the agency imposed last December after a case of persistent anemia, helps clear the way for Bluebird to submit an approval application early next year. 

By Ned Pagliarulo • Dec. 19, 2022

Sio Gene Therapies to dissolve after years of setbacks

Having failed to find a buyer or potential partner, the biotech company has opted instead to shut down and liquidate its assets.

By Delilah Alvarado • Dec. 15, 2022

Roche’s hemophilia gene therapy holds steady with longer-term data

Roche’s subsidiary Spark has offered few updates on its hemophilia A treatment since being acquired in 2019. New data at ASH show the therapy can maintain levels of a key blood-clotting protein for years. 

By Jonathan Gardner • Dec. 12, 2022

Trailing rivals, Editas shares first study data for sickle cell treatment

Editas said its gene editing therapy appeared safe and was well tolerated in two patients. The data comes five months after U.S. regulators lifted a partial hold on the trial.

By Kristin Jensen • Dec. 7, 2022

Gene therapy approvals bring validation as field closes year on high

It’s been a busy fall in gene therapy, with FDA nods for new treatments from CSL and Bluebird as well as a few deals. Catch up on our best stories here. 

By Ned Pagliarulo • Dec. 6, 2022

Verve shares slide after company reveals details on FDA trial hold

In a letter to the biotech, the regulator asked for more information on its gene editing medicine for heart disease as well as on its potential risks.

By Delilah Alvarado • Dec. 6, 2022