Gene Therapy - Clinical Research Informer

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

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.

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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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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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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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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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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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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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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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‘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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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.

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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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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.

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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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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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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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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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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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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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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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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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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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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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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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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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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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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.

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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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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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