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

Explore the Trendline➔

The BioPharma Dive Outlook on 2023

Against a stormy backdrop, 2022 did bring bright spots in Alzheimer’s, obesity, breast cancer and rare disease. Dig in to BioPharma Dive’s 2023 outlook for what’s in store this year. 

By BioPharma Dive staff

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

FDA lifts pause on Beam plans to test base editing cancer therapy

The FDA’s decision is a step forward for a type of drugmaking known as “multiplex editing,” which involves editing several genes simultaneously.

By Ben Fidler • Dec. 2, 2022

Gene editing startup iECURE returns to investors for fresh funding

The company, which draws on research by Jim Wilson’s lab at UPenn, says the $65 million it raised will support the start of human testing of its lead drug through to initial clinical data.

By Gwendolyn Wu • Nov. 30, 2022

AstraZeneca reveals its cell therapy ambitions with deal for startup

The British drugmaker will pay as much as $320 million to acquire Neogene Therapeutics, a developer of cell-based treatments for solid tumors — its first significant investment in the field.

By Ben Fidler • Nov. 29, 2022

FDA grants speedy review to Sarepta’s Duchenne gene therapy

The agency will decide on an accelerated approval of Sarepta’s treatment by May 29, months before results are expected from a potentially confirmatory Phase 3 trial.

By Jonathan Gardner • Nov. 28, 2022

Navigate the complexities of cell and gene therapies

See how the effective use of CMC expertise helped guide a biopharma company’s development of a CGT product.

Nov. 28, 2022

Mainstreaming cell and gene therapy – Realizing its potential

The challenges biopharma companies face in running clinical trials and tips on how to surmount those obstacles.

Nov. 28, 2022

FDA approves first gene therapy for hemophilia B

The one-time treatment, which is for the less common “B” form of the bleeding disorder, will be sold in the U.S. by maker CSL for $3.5 million per infusion. 

By Ned Pagliarulo • Updated Nov. 23, 2022

Editas to seek partner for CRISPR medicine after lackluster study results

While the biotech will not develop the gene editing therapy further on its own, CEO Gilmore O’Neill claimed the data are still a meaningful demonstration of what CRISPR can accomplish. 

By Ned Pagliarulo • Nov. 17, 2022

Ionis teams with Metagenomi and dives into gene editing

The RNA drug developer is paying the richly funded startup $80 million in a wide-ranging alliance that marks its first foray into DNA editing medicines.

By Ben Fidler • Nov. 14, 2022