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.
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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.
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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.
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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.
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Making the Case for Case Management for Cell and Gene Therapies
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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.
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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.
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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.
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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.
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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.
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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.
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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.
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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.
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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.
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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.
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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.
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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.
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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.
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‘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.
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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.
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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.
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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.
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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.
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Kebilidi FDA-Approved as First Brain-Delivered Gene Therapy for AADC Deficiency
XTalks
NOVEMBER 14, 2024
PTC Therapeutics has gained US Food and Drug Administration (FDA) approval for its new gene therapy, Kebilidi (eladocagene exuparvovec), for treating aromatic L-amino acid decarboxylase (AADC) deficiency.
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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.
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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.
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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.
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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.
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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.
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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.
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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.
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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.
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Pfizer shelves haemophilia gene therapy Beqvez amid low demand
Pharmaceutical Technology
FEBRUARY 21, 2025
Pfizer said resources will be directed to its other haemophilia gene therapy Hympavzi.
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MeiraGTx and Hologen launch AI-backed gene therapy venture
Pharmaceutical Technology
MARCH 14, 2025
The venture aims to advance MeiraGTxs Parkinsons gene therapy through Phase III trials with up to $430m in funding from Hologen.
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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.
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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.
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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.”
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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.
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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.
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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.
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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.
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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.
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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.
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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.
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