Gene - 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.

Gene Therapy

Gene Therapy Gene Research

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

Gene Therapy Gene Development

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

Gene Therapy Gene

Pfizer expands into gene editing with Beam research deal

Bio Pharma Dive

JANUARY 10, 2022

Pfizer's work with mRNA vaccines led it explore other applications of the technology, resulting in a multi-year partnership with the high-profile biotech Beam Therapeutics on gene editing treatments for rare diseases.

Gene Editing

Gene Editing Gene Research Vaccination

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.

Gene Therapy

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

Gene Therapy Gene

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

Gene Therapy Gene Marketing Research

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

Gene Therapy Gene Development Genetics

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

Gene Therapy Gene Drugs

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

Gene Therapy Gene Scientist Clinical Trials

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

Gene Therapy Gene Drugs

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

Gene Therapy Gene

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.

Gene Therapy

Gene Therapy Gene Trials

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

Gene Therapy Gene Development

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

Gene Therapy Gene

‘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

Gene Therapy Gene Development

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

Gene Therapy Gene Drugs

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

Gene Therapy Gene

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

Gene Therapy Gene

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

Gene Therapy Manufacturing Gene

Sarepta’s Gene Therapy Elevidys Under Scrutiny After Patient Death

XTalks

MARCH 19, 2025

A 16-year-old patient died after treatment with Elevidys (delandistrogene moxeparvovec), Sarepta Therapeutics gene therapy for Duchenne muscular dystrophy (DMD). Liver injury is a known risk with Elevidys and other gene therapies that use adeno-associated viral (AAV) vectors. Sarepta is reviewing all available data.

Gene Therapy

Gene Therapy Gene Genetics Clinical Trials

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

Gene Therapy Gene Trials

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

Gene Therapy Manufacturing Gene Medicine

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.

Gene Therapy

Gene Therapy Gene

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 Expression

Gene Expression Gene Development

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

Gene Therapy Gene

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

Bio Pharma Dive

MARCH 2, 2023

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

Gene Editing

Gene Editing In-Vivo Gene Drugs

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

Gene Therapy Gene

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

Gene Therapy Gene

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

Gene Therapy Gene Research

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

Gene Therapy Gene

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

Gene Therapy Gene Marketing

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

Gene Therapy Gene Contract Manufacturing Manufacturing

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

Gene Therapy Gene

The future of cell and gene therapy manufacturing

Pharmaceutical Technology

NOVEMBER 30, 2023

As more cell and gene therapies get approved to treat common conditions, scaling them up will be a challenge.

Gene Therapy

Gene Therapy Gene Manufacturing

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

Gene Therapy Gene FDA Approval Drugs

Duchenne patient dies after receiving Sarepta gene therapy

Bio Pharma Dive

MARCH 18, 2025

A young man treated with Elevidys died of acute liver failure. The case may give doctors pause before prescribing the treatment in the future, some analysts wrote.

Gene Therapy

Gene Therapy Gene Doctors Doctor

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

Gene Therapy Gene Marketing

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

Gene Gene Therapy

A startup launches with plans to open up a gene and cell therapy bottleneck

Bio Pharma Dive

JANUARY 31, 2023

The pipeline of CAR-T therapies and ex vivo gene therapies has swelled in recent years, but manufacturing hasn’t been able to keep up with demand.

Gene

Gene In-Vivo Gene Therapy Manufacturing

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

Gene Therapy Gene

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

Gene Editing Gene Development Medicine

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

Gene Therapy Gene Drugs

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

Gene Therapy Gene Drugs

5 questions facing gene therapy in 2022

Cell and gene therapy investment, once booming, is now in a slump

Trending Sources

Duchenne approval exposes FDA rift over Sarepta gene therapy

Pfizer expands into gene editing with Beam research deal

Making the Case for Case Management for Cell and Gene Therapies

FDA endorses speedy approval path for Regenxbio Duchenne gene therapy

As biotech retreats, gene therapy companies retrench and redraw plans

Cyagen and Neurophth partner to develop gene therapy vectors

FDA halts tests of BioMarin drug amid heightened focus on gene therapy safety

Scientists home in on cause of Duchenne gene therapy side effect

Pfizer hemophilia gene therapy arrives in US to uncertain future

Gene therapy startup Vedere to close two years after launch

MeiraGTx and Hologen launch AI-backed gene therapy venture

Prevail, Capsida partner to develop gene therapies for nervous system diseases

Lexeo gene therapy shows signs of heart benefit in small study

‘We have to find a way’: FDA seeks solutions to aid bespoke gene therapy

Bispecific cancer drugs and gene therapy advances: What to watch at next month’s ASH meeting

What’s next for AAV gene therapies in 2024?

With Duchenne decision ahead, FDA’s Marks pushes for speedy gene therapy approvals

Cell and gene therapy approvals drive paradigm change in manufacturing

Sarepta’s Gene Therapy Elevidys Under Scrutiny After Patient Death

Pfizer gene therapy for Duchenne fails to meet goals of key trial

Gene therapy biotech Jaguar spins out manufacturing company

Pfizer shelves haemophilia gene therapy Beqvez amid low demand

Reducing variability in gene expression: bottlenecks and solutions

Medicaid, with planned payment pilot, girds for influx of pricey gene therapies

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

Why are haemophilia gene therapies so expensive?

Freeline cuts more jobs and halts Fabry gene therapy work

Siren emerges from stealth with plans to marry gene therapy to cancer immunotherapy

Streamlining IND applications for cell and gene therapy innovations

Cell and gene therapy companies trip at scalability hurdle

Ajinomoto spices up its biopharma business with gene therapy deal

CRISPR gene therapy Casgevy secures approval in Europe

The future of cell and gene therapy manufacturing

Pfizer receives FDA approval for haemophilia B gene therapy

Duchenne patient dies after receiving Sarepta gene therapy

Orchard sells to Kyowa Kirin in gene therapy buyout

Lexeo wins fast track designation for Friedreich’s ataxia gene therapy

A startup launches with plans to open up a gene and cell therapy bottleneck

An Italian biotech gets $52M to advance pediatric gene therapies

Editas and Genevant team up to develop gene editing therapies

FDA grants accelerated approval for PTC’s AADC deficiency gene therapy

AskBio’s AB-1002 gene therapy receives FDA fast-track status

Stay Connected