Gene Therapy
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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➔
Spencer Platt via Getty ImagesTrendlineThe 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 -
Q&A
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 -
Gene therapy safety
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 -
Gene editing
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 -
Gene therapy safety
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 -
Gene editing
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
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 -
Sponsored by Cardinal Health
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 -
Sponsored by ICON
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 -
Gene editing
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 -
Gene editing
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