Reducing variability in gene expression: bottlenecks and solutions
Bio Pharma Dive
NOVEMBER 18, 2024
Explore solutions to reduce variability in gene expression during cell line development.
Gene Related Topics 
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 
215
215 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 356
356 
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 
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.
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 334
334 
Editas and Genevant team up to develop gene editing therapies
Pharmaceutical Technology
OCTOBER 22, 2024
Editas is partnering its CRISPR platform with Genevant’s LNP technology to develop novel gene editing medicines in a deal worth $283m.
Gene Editing 244
244 
An Italian biotech gets $52M to advance pediatric gene therapies
Bio Pharma Dive
SEPTEMBER 25, 2024
The financing will support a technology designed to make delivery of lentiviral gene therapies, which are typically administered through a complex process, more convenient.
Gene Therapy 246
246 
ReCode to secure funds for cystic fibrosis gene correction treatments
Pharmaceutical Technology
NOVEMBER 19, 2024
ReCode is set to secure up to $15m in funding from the Cystic Fibrosis Foundation to enhance its gene correction research programme.
Gene 130
130 
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 
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 
FDA grants accelerated approval for PTC’s AADC deficiency gene therapy
Pharmaceutical Technology
NOVEMBER 14, 2024
The US Food and Drug Administration (FDA) has granted accelerated approval for PTC Therapeutics’ KEBILIDI, the first gene therapy in the US that is directly delivered to the brain.
Gene Therapy 130
130 
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 298
298 
PTC wins US approval of gene therapy for fatal enzyme disorder
Bio Pharma Dive
NOVEMBER 14, 2024
Kebilidi is the first approved gene therapy that can be directly administered to the brain. Its OK secures a priority review voucher for PTC.
Gene Therapy 141
141 
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 286
286 
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 294
294 
Dyno and Roche collaborate on neurological gene therapies
Pharmaceutical Technology
OCTOBER 25, 2024
Dyno has formed a new research partnership with Roche on the development of AAV gene therapy vectors for neurological diseases.
Gene Therapy 147
147 
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 278
278 
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 
AskBio’s AB-1002 gene therapy receives FDA fast-track status
Pharmaceutical Technology
APRIL 19, 2024
The US Food and Drug Administration (FDA) has granted fast-track designation to Bayer subsidiary Asklepios BioPharmaceutical’s (AskBio) investigational gene therapy AB-1002, aimed at treating congestive heart failure (CHF).
Gene Therapy 245
245 
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 
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 receives FDA approval for haemophilia B gene therapy
Pharmaceutical Technology
APRIL 29, 2024
Pfizer has received approval from the US Food and Drug Administration (FDA) for its one-time gene therapy BEQVEZ for haemophilia B.
Gene Therapy 245
245 
Scientists Just Identified Hundreds of Genes That Could Cause Cancer
AuroBlog - Aurous Healthcare Clinical Trials blog
SEPTEMBER 22, 2024
(Richard Jones/Science Photo Library/Getty Images) Scientists have discovered hundreds of genes that could potentially promote cancer, new research shows. Cancer is typically triggered by some kind of change to our genetic coding that interferes with a cell’s ability to manage its growth.
Scientist 179
179 
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 278
278 
Jim Wilson, prominent gene therapy researcher, to depart UPenn
Bio Pharma Dive
AUGUST 1, 2024
Wilson, who founded UPenn’s gene therapy program three decades ago, will step down to start two new spinouts, Gemma Bio and Franklin Biolabs.
Gene Therapy 183
183 
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 
Pfizer setback brings questions for Duchenne gene therapy ahead of Sarepta decision
Bio Pharma Dive
JUNE 13, 2024
The failure of another trial adds to uncertainty around the benefits of the gene-based treatments, though industry analysts still expect Sarepta’s Elevidys’ to be unaffected.
Gene 240
240 
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 
Lexeo wins fast track designation for Friedreich’s ataxia gene therapy
Pharmaceutical Technology
APRIL 17, 2024
LX2006 consists of adeno-associated virus (AAV) vector carrying the frataxin gene, responsible for causing Friedreich’s ataxia.
Gene 246
246 
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 268
268 
Delivery tech remains challenging with gene therapies for brain disorders
Pharmaceutical Technology
MARCH 21, 2024
Biotech executives discuss why developing gene therapies for central nervous system disorders remains difficult at a recent conference.
Gene Therapy 246
246 
Genevoyager opens CDMO facility for gene therapy development
Pharmaceutical Technology
DECEMBER 21, 2023
Genevoyager has announced the opening of contract development and manufacturing organisation facility to manufacture gene therapy products.
Gene Therapy 243
243 
Anocca signs licensing deal for EmendoBio’s gene-editing tech
Pharmaceutical Technology
MARCH 15, 2024
Anocca has entered a licensing agreement with EmendoBio for the use of the latter’s OMNI-A4 nuclease, a gene editing technology.
Gene Editing 244
244 
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 328
328 
Are haemophilia gene therapies truly “one and done”?
Pharmaceutical Technology
NOVEMBER 20, 2023
The value of haemophilia gene therapies like Roctavian and Hemgenix hinge on long-term efficacy and innovative payment structures play a key role.
Gene Therapy 246
246 
Lexeo gene therapy shows signs of heart benefit in small study
Bio Pharma Dive
JULY 15, 2024
The company claims the results support exploring the possibility of an accelerated approval, citing flexibility by FDA officials in reviewing rare disease gene therapies.
Gene Therapy 183
183 
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 270
270 
PTC wins FDA approval for first brain-delivered gene therapy Kebilidi
Fierce Pharma
NOVEMBER 14, 2024
The FDA issued its first stamp of approval for a cell or gene therapy back in 2017 to Novartis' Kymriah. | The gene therapy has already been approved to treat AADC deficiency in Europe and the U.K., among other countries, where it's branded as Upstaza.
Gene Therapy 114
114 
Ocuphire and Opus Genetics merge to develop IRD gene therapy
Pharmaceutical Technology
OCTOBER 23, 2024
Ocuphire Pharma has announced the acquisition of Opus Genetics to develop gene therapies for inherited retinal diseases (IRDs).
Gene Therapy 130
130 
Regeneron gene therapy improves hearing in two children
Bio Pharma Dive
MAY 8, 2024
The results add to an early but growing body of evidence suggesting gene therapy may improve outcomes in young kids with a genetic form of deafness.
Gene Therapy 195
195 
Ophthalmologists eye gene therapies for vision-threatening conditions as NGS facilitates precision diagnosis
AuroBlog - Aurous Healthcare Clinical Trials blog
SEPTEMBER 9, 2024
The convergence of gene therapies and clinical research is pushing the boundaries of what’s possible in ophthalmic care, offering hope for more effective treatments and potential cures for a range of vision-threatening conditions.
Gene Therapy 200
200 
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.
Gene Therapy 352
352 
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 268
268 
Let's personalize your content