Moderna inks second genetic medicine deal in a month

The biotech plans to use Generation Bio’s nonviral delivery technology to get nucleic acid-based therapies to immune cells and into the liver.

By Christopher Newman • March 23, 2023

Gilead’s Yescarta extends survival in lymphoma study

The results, the first for a CAR-T therapy, are further evidence supporting the use of the complex therapies earlier in a patient’s disease course. 

By Ben Fidler • March 21, 2023

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

Addressing manufacturing challenges in gene therapy development

As new gene therapies are developed to treat a growing number of indications, patients may soon have access to novel treatment options and potential cures.

By Kate Silver • March 20, 2023

Switch Therapeutics launches with $52M to capitalize on RNA medicine ‘era’

The company’s name comes from its goal of developing drugs that only “switch” on in target cells, potentially enabling more precise therapies.

By Gwendolyn Wu • March 14, 2023

BioMarin, as expected, sees delay to FDA review of hemophilia gene therapy

The agency is considering three-year follow-up data in its review of Roctavian, pushing back the potential U.S. arrival of BioMarin’s drug once again.

By Kristin Jensen • March 7, 2023

Voyager licenses gene therapy tools to Novartis

The Swiss drugmaker's decision is the latest in a string of partnership announcements for the gene therapy maker, which aims to bounce back from past research failures.

By Ben Fidler • March 6, 2023

FDA clears Intellia to start US tests of ‘in vivo’ gene editing drug

The announcement, which follows regulatory setbacks for some of Intellia’s peers, is a “big win” for the gene editing field, according to one analyst.

By Ben Fidler • March 2, 2023

Amid industry turbulence, Chroma raises fresh funding to edit the epigenome

As many drug startups struggle to gain cash, Chroma has raised another $135 million to back its research, a new twist on genetic medicine.

By Gwendolyn Wu • Updated March 1, 2023

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

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