5 questions facing gene therapy in 2022
Bio Pharma Dive
JANUARY 13, 2022
As record levels of money pour into gene therapy research, biotechs are under increased pressure to address issues related to safety, delivery and overlapping pipelines.
Duchenne approval exposes FDA rift over Sarepta gene therapy
Bio Pharma Dive
JUNE 21, 2024
Peter Marks’ decision to override the objections of agency staff and broaden use of Elevidys could have a “lasting impact” on gene therapy as well as the FDA, one analyst wrote.
Gene Therapy 
338
338 Join 21,000+
Insiders
Sign Up for our Newsletter
This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.
Cell and gene therapy investment, once booming, is now in a slump
Bio Pharma Dive
AUGUST 29, 2024
Far fewer venture funding rounds were closed by cell and gene therapy developers over the first six months of 2024 than in prior years. Experts say there are several factors.
Gene Therapy 357
357 
FDA endorses speedy approval path for Regenxbio Duchenne gene therapy
Bio Pharma Dive
NOVEMBER 18, 2024
The agency's openness to a targeted pivotal study shows it’s still willing to consider accelerated clearance for Duchenne gene therapies despite questions about their effectiveness.
Gene Therapy 336
336 
Making the Case for Case Management for Cell and Gene Therapies
Advertisement
As demand for advanced therapies increases, so does the need for more specialized supply chain support, as these products have strict transportation and handling requirements.
At FDA meeting, gene therapy experts wrestle with field's blindspots
Bio Pharma Dive
SEPTEMBER 3, 2021
A panel convened by the regulator suggested ways to make gene therapy research safer, but struggled to propose broader recommendations for the field.
Gene Therapy 362
362 
Gene therapy for Fabry: early stages, promising results
Bio Pharma Dive
JULY 22, 2021
Three companies are testing their gene therapies in early clinical trials, with initial results due later this year. Others are in preclinical stages and aim to follow soon.
Gene Therapy 347
347 
As biotech retreats, gene therapy companies retrench and redraw plans
Bio Pharma Dive
MARCH 24, 2022
At least nine biotechs working in cell or gene therapy have announced layoffs, cost cuts or restructured their research since December — restructurings that have coincided with a stock market downturn.
Gene Therapy 361
361 
A high-profile gene therapy biotech takes aim at Huntington's
Bio Pharma Dive
DECEMBER 8, 2021
Passage Bio and the University of Pennsylvania's Gene Therapy Program said they have begun preclinical research aimed at simultaneously developing multiple approaches to treating Huntington's.
Gene Therapy 347
347 
What’s next for AAV gene therapies in 2024?
Pharmaceutical Technology
MARCH 11, 2024
Upcoming regulatory events for pipeline AAV gene therapies, with a focus on neuromuscular and blood disorders, are setting high expectations for the space in 2024.
Gene Therapy 299
299 
Pfizer hemophilia gene therapy arrives in US to uncertain future
Bio Pharma Dive
APRIL 26, 2024
The Food and Drug Administration approval of Beqvez comes as other gene therapies for the bleeding condition that were approved earlier struggle to gain traction.
Gene Therapy 310
310 
Cyagen and Neurophth partner to develop gene therapy vectors
Pharmaceutical Technology
NOVEMBER 8, 2022
Cyagen and Neurophth Therapeutics have entered a strategic partnership to jointly develop next-generation AAV gene therapy vectors for specific kinds of genetic ophthalmic ailments. Additionally, Neurophth will oversee the clinical trials and marketing of gene therapy products developed leveraging the new AAV capsids of Cyagen.
Gene Therapy 305
305 
FDA halts tests of BioMarin drug amid heightened focus on gene therapy safety
Bio Pharma Dive
SEPTEMBER 7, 2021
On the heels of a two-day FDA meeting in which experts debated the risks of gene therapy, the biotech said its early-stage PKU treatment was associated with cancer in mice.
Gene Therapy 362
362 
Scientists home in on cause of Duchenne gene therapy side effect
Bio Pharma Dive
MAY 17, 2022
An unusual collaboration among gene therapy developers suggests certain mutations could be behind "peculiar" side effects experienced by several patients treated in clinical trials.
Gene Therapy 344
344 
Gene therapy biotech Jaguar spins out manufacturing company
Bio Pharma Dive
JANUARY 18, 2024
Called Advanced Medicine Partners, the new company will offer specialized cell and gene therapy manufacturing services to biotech and pharma clients.
Gene Therapy 290
290 
Gene therapy startup Vedere to close two years after launch
Bio Pharma Dive
APRIL 2, 2023
The startup, a successor to an eye gene therapy biotech that Novartis bought in 2020, will wind down after preclinical experiments didn’t meet the bar set by its leaders.
Gene Therapy 334
334 
What to expect at the FDA's two-day meeting on gene therapy safety
Bio Pharma Dive
SEPTEMBER 1, 2021
A group of advisers convened by the regulator will spend Thursday and Friday weighing a range of safety worries with gene therapy, and how best to prevent them.
Gene Therapy 349
349 
Prevail, Capsida partner to develop gene therapies for nervous system diseases
Bio Pharma Dive
JANUARY 4, 2023
The Lilly-owned subsidiary will pay $55 million to gain access to Capsida’s AAV gene therapy technology in a deal aimed at central nervous system disorders.
Gene Therapy 334
334 
A biotech startup raises $117M to deliver gene therapies in a new way
Bio Pharma Dive
JULY 28, 2021
Flagship Pioneering, the biotech incubator that created Moderna, is helping fund Ring Therapeutics, a startup hoping to disrupt gene therapy development through the use of a different viral vector.
Gene Therapy 345
345 
With Duchenne decision ahead, FDA’s Marks pushes for speedy gene therapy approvals
Bio Pharma Dive
MAY 24, 2024
But the head of the FDA’s CBER office didn’t tip where the agency stands on potentially broadening use of Sarepta’s Duchenne gene therapy Elevidys.
Gene Therapy 306
306 
‘We have to find a way’: FDA seeks solutions to aid bespoke gene therapy
Bio Pharma Dive
OCTOBER 13, 2022
Gene therapies could help treat many ultra-rare diseases. But they may not get developed if drugmakers can’t build a sustainable business around them, CBER director Peter Marks said at a conference.
Gene Therapy 347
347 
Astellas takes stake in gene therapy developer Taysha
Bio Pharma Dive
OCTOBER 24, 2022
Already an investor in gene therapy, the Japanese pharma will buy 15% of the Texas biotech’s stock and gain options to two gene therapies for central nervous system disorders.
Gene Therapy 282
282 
Bispecific cancer drugs and gene therapy advances: What to watch at next month’s ASH meeting
Bio Pharma Dive
NOVEMBER 4, 2022
Competition is increasing behind newly approved drugs from Roche and J&J, while the long-term benefit of gene therapies for chronic blood disorders is being put to the test.
Gene Therapy 348
348 
Lilly agrees to acquire gene therapy developer Akouos for $610m
Pharmaceutical Technology
OCTOBER 19, 2022
Akouos focuses on developing adeno-associated viral gene therapies to treat inner ear conditions, including sensorineural hearing loss. It has combined expertise in otology, gene therapy and inner ear drug delivery to meet the requirements of individuals with disabling hearing loss globally.
Gene Therapy 298
298 
Pfizer gene therapy for Duchenne fails to meet goals of key trial
Bio Pharma Dive
JUNE 12, 2024
The negative results from the Phase 3 study, called Ciffreo, come a little more than a month after Pfizer said a patient died in another trial of the gene therapy.
Gene Therapy 299
299 
Roche digs deeper into gene therapy for the eye
Bio Pharma Dive
JULY 20, 2022
The Swiss drugmaker, which already owns rights to a marketed gene therapy for inherited vision loss, will work with startup Avista Therapeutics to develop better delivery tools for the complex treatments.
Gene Therapy 306
306 
BioMarin's hemophilia gene therapy recommended for approval in Europe
Bio Pharma Dive
JUNE 24, 2022
The decision brings what could be the first approved hemophilia gene therapy, Roctavian, closer to market, after a series of regulatory setbacks that have delayed its arrival.
Gene Therapy 304
304 
Cell and gene therapy approvals drive paradigm change in manufacturing
Pharmaceutical Technology
OCTOBER 14, 2024
Trends in gene therapy approvals in recent years are rapidly shaping the future of manufacturing capabilities in the pharmaceutical industry.
Gene Therapy 279
279 
CRISPR gene therapy Casgevy secures approval in Europe
Pharmaceutical Technology
FEBRUARY 13, 2024
The regulatory greenlight makes it the only gene therapy available in Europe for sickle cell disease and TDT.
Gene Therapy 264
264 
Years later, a first-of-its-kind treatment shows the power, and limits, of gene therapy
Bio Pharma Dive
NOVEMBER 15, 2021
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.
Gene Therapy 345
345 
Voyager licenses gene therapy tools to Novartis
Bio Pharma Dive
MARCH 6, 2023
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.
Gene Therapy 280
280 
Medicaid, with planned payment pilot, girds for influx of pricey gene therapies
Bio Pharma Dive
FEBRUARY 21, 2023
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.
Gene Therapy 312
312 
Another Astellas gene therapy trial paused by FDA after side effect report
Bio Pharma Dive
JUNE 27, 2022
Both of the pharma’s clinical-stage gene therapies are now on hold. The regulator suspended testing of Astellas’ Pompe disease treatment after one patient experienced peripheral nerve damage.
Gene Therapy 311
311 
Freeline cuts more jobs and halts Fabry gene therapy work
Bio Pharma Dive
APRIL 4, 2023
biotech will lay off 30% of its workforce and focus on an experimental Gaucher disease treatment, five months after an initial restructuring that cut a hemophilia gene therapy.
Gene Therapy 295
295 
Ajinomoto spices up its biopharma business with gene therapy deal
Bio Pharma Dive
NOVEMBER 13, 2023
Forge Biologics operates a contract manufacturing business as well as develops its own gene therapies, which appears to have attracted the Japan-based food and biotechnology company.
Gene Therapy 290
290 
Siren emerges from stealth with plans to marry gene therapy to cancer immunotherapy
Bio Pharma Dive
MAY 17, 2023
Spun out of the lab of former UCSF researcher Nicole Paulk, the biotech is targeting brain and eye tumors with a new kind of treatment it has dubbed “immuno-gene therapy.”
Gene Therapy 310
310 
Pharma partner Sartorius eyes gene therapy market with $2.6B deal
Bio Pharma Dive
MARCH 31, 2023
The biotech tools supplier is acquiring Polyplus, a French company that makes components essential to the viral vector backbones of gene therapies.
Gene Therapy 285
285 
Why are haemophilia gene therapies so expensive?
Pharmaceutical Technology
OCTOBER 11, 2023
Biomarin and CSL Behring’s haemophilia gene therapies have boosted innovation in the field, but assessing their cost effectiveness is less than straightforward.
Gene Therapy 264
264 
Orchard sells to Kyowa Kirin in gene therapy buyout
Bio Pharma Dive
OCTOBER 5, 2023
Kyowa will pay nearly $400 million to acquire Orchard, which sells the gene therapy Libmeldy in Europe but has struggled to find paths to market for other experimental treatments.
Gene Therapy 278
278 
Streamlining IND applications for cell and gene therapy innovations
Bio Pharma Dive
SEPTEMBER 23, 2024
Regulatory and strategic challenges can delay the delivery of life-changing cell and gene therapies.
Gene Therapy 246
246 
Forge Biologics and Labcorp partner for gene therapies development
Pharmaceutical Technology
MAY 4, 2023
Forge Biologics and global life sciences company Labcorp have announced a strategic adeno-associated virus (AAV) gene therapy development and manufacturing collaboration. This will improve accessibility to services for AAV-mediated gene therapy programmes.
Gene Therapy 256
256 
Cell and gene therapy companies trip at scalability hurdle
Pharmaceutical Technology
APRIL 29, 2024
Experts hold scalability challenges and high costs accountable for market failures within the cell and gene therapy landscape.
Gene Therapy 246
246 
Pfizer’s hemophilia B gene therapy inches closer to regulatory approval
Bio Pharma Dive
JUNE 27, 2023
The treatment could become Pfizer’s first marketed gene therapy, an area the pharma has poured significant resources into in recent years.
Gene Therapy 279
279 
FDA fast tracks Solid Biosciences’s Duchenne gene therapy
Pharmaceutical Technology
DECEMBER 7, 2023
The FDA has granted fast track designation to Solid Biosciences’s gene therapy SGT-003 for the treatment of DMD.
Gene Therapy 264
264 
Let's personalize your content