Gene Therapy - Clinical Research Informer

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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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

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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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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

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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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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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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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.

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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).

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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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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.

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Gene Therapy Gene Genetics

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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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.

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Gene Therapy Gene Development

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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Q&A: Cell and gene therapy value assessments need a rework to allow access

Pharmaceutical Technology

APRIL 19, 2024

Paolo Morgese, vice president of European public affairs at the Alliance for Regenerative Medicine, discusses the barriers affecting access to gene therapies.

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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

Gene Therapy Gene Development Manufacturing

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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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.

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How cell and gene therapy are evolving

Pharmaceutical Technology

MAY 30, 2024

The cell and gene therapy market is accelerating in its evolution, as well as commercial approvals, with new therapeutic options for patients

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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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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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Medicines access: Unlocking the immense potential in cell and gene therapy

Pharmaceutical Technology

FEBRUARY 8, 2024

Despite several ground-breaking developments, pharmaceutical and biotech companies are often reluctant to invest in cell and gene therapy.

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Gene Therapy Gene Medicine Development

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

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

Gene Therapy Gene Development Manufacturing

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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The role of a specialty pharmacy in enhancing patient access to rare and gene therapies

Bio Pharma Dive

JUNE 24, 2024

Four critical components in which the right specialty pharmacy partner can bring rare and gene therapy manufacturers the scale, expertise and capabilities needed to drive enhanced patient access.

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Spark Therapeutics partners with SpliceBio for gene therapy

Pharmaceutical Technology

OCTOBER 17, 2023

SpliceBio will license Spark Therapeutics’ propriety protein splicing platform to develop a gene therapy for renal disease.

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Cell and gene therapies – top pharmaceutical industry trend to watch for 2024

Pharmaceutical Technology

JANUARY 29, 2024

The full potential of cell and gene therapies (CGTs) has not yet been discovered as these therapies promise life-changing treatments.

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Avrobio to sell cystinosis gene therapy to Novartis for $87.5m

Pharmaceutical Technology

MAY 23, 2023

Avrobio has announced a deal to sell its investigational haematopoietic stem cell (HSC) gene therapy programme , designed to treat cystinosis, to Novartis in an all-cash deal valued at $87.5m. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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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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How gene therapies can transform sickle cell disease treatment

Pharmaceutical Technology

OCTOBER 26, 2023

The potential for using gene therapies to treat sickle cell disease is high, but their durability and high price point invite scrutiny.

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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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Experts Believe Gene Therapy Offers Potential to Impact Gaucher Disease Market

Pharmaceutical Technology

NOVEMBER 21, 2023

According to several key opinion leaders (KOLs) interviewed by GlobalData, pipeline gene therapy holds the greatest potential to transform the Gaucher disease landscape

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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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Gene Therapy Gene Research

Robotics: Transforming cell and gene therapy manufacturing

Pharmaceutical Technology

AUGUST 16, 2023

Several companies are developing robotics to automate cell and gene therapy manufacturing, but some challenges persist.

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Gene Therapy Manufacturing Gene Development

First gene therapies for sickle cell disease secure FDA approval

Pharmaceutical Technology

DECEMBER 11, 2023

The FDA has approved Vertex/CRISPR’s Casgevy and bluebird bio’s Lyfgenia gene therapies for the red blood cell disorder.

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FDA Rejects Rocket’s Gene Therapy, Cites Need for Additional CMC Info

BioSpace

JUNE 27, 2024

Rocket Pharmaceuticals’ gene therapy Kresladi has been hit with an FDA Complete Response Letter requesting additional chemistry, manufacturing and controls information to complete its review.

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Gene Therapy Gene Manufacturing

Astellas to support development of Taysha’s gene therapy programmes

Pharmaceutical Technology

OCTOBER 25, 2022

Ast ellas Pharma has announced plans to make a strategic investment to back the development of Taysha Gene Therapies’ adeno-associated virus (AAV) development programmes for Rett syndrome and giant axonal neuropathy (GAN). Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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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.

Gene Therapy

Gene Therapy Gene Development Genetics

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

Addressing manufacturing challenges in gene therapy development

Bio Pharma Dive

MARCH 20, 2023

As new gene therapies are developed to treat a growing number of indications, patients may soon have access to novel treatment options and potential cures.

Gene Therapy

Gene Therapy Gene Development Manufacturing

Beacon Therapeutics enters gene therapy arena with $120m launch

Pharmaceutical Technology

JUNE 13, 2023

Beacon Therapeutics has kickstarted its entry into the gene therapy field with a $120m Series A financing. Amongst it was AGTC’s lead clinical candidate, AFTC-501, an adeno-associated virus (AAV) gene therapy for XLRP. Beacon Therapeutics is the third gene therapy company Syncona has launched.

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4D Molecular’s Fabry gene therapy program on FDA clinical hold

Pharmaceutical Technology

FEBRUARY 10, 2023

4D Molecular Therapeutics (4DMT), the California-based biotechnology company focused on developing gene therapies for rare and large market diseases, has had the FDA place a clinical hold onto its Fabry disease (FD) gene therapy program (4D-310).

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Sarepta offers early look at closely watched gene therapy launch

Bio Pharma Dive

AUGUST 3, 2023

One patient received its Duchenne gene therapy, Elevidys, this week and a handful more could follow shortly, surprising some analysts and raising near-term sales expectations.

Gene Therapy

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Duchenne approval exposes FDA rift over Sarepta gene therapy

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

Trending Sources

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

Pfizer hemophilia gene therapy arrives in US to uncertain future

Making the Case for Case Management for Cell and Gene Therapies

What’s next for AAV gene therapies in 2024?

Cell and gene therapy companies trip at scalability hurdle

Pfizer receives FDA approval for haemophilia B gene therapy

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

Gene therapy biotech Jaguar spins out manufacturing company

Regeneron gene therapy improves hearing in two children

Why are haemophilia gene therapies so expensive?

Delivery tech remains challenging with gene therapies for brain disorders

CRISPR gene therapy Casgevy secures approval in Europe

Q&A: Cell and gene therapy value assessments need a rework to allow access

Genevoyager opens CDMO facility for gene therapy development

Ajinomoto spices up its biopharma business with gene therapy deal

Are haemophilia gene therapies truly “one and done”?

How cell and gene therapy are evolving

FDA fast tracks Solid Biosciences’s Duchenne gene therapy

Orchard sells to Kyowa Kirin in gene therapy buyout

Medicines access: Unlocking the immense potential in cell and gene therapy

The future of cell and gene therapy manufacturing

Forge Biologics and Labcorp partner for gene therapies development

Gene therapy startup Vedere to close two years after launch

The role of a specialty pharmacy in enhancing patient access to rare and gene therapies

Spark Therapeutics partners with SpliceBio for gene therapy

Cell and gene therapies – top pharmaceutical industry trend to watch for 2024

Avrobio to sell cystinosis gene therapy to Novartis for $87.5m

Astellas takes stake in gene therapy developer Taysha

How gene therapies can transform sickle cell disease treatment

Voyager licenses gene therapy tools to Novartis

Experts Believe Gene Therapy Offers Potential to Impact Gaucher Disease Market

5 questions facing gene therapy in 2022

Robotics: Transforming cell and gene therapy manufacturing

First gene therapies for sickle cell disease secure FDA approval

FDA Rejects Rocket’s Gene Therapy, Cites Need for Additional CMC Info

Astellas to support development of Taysha’s gene therapy programmes

Lilly agrees to acquire gene therapy developer Akouos for $610m

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

Addressing manufacturing challenges in gene therapy development

Beacon Therapeutics enters gene therapy arena with $120m launch

4D Molecular’s Fabry gene therapy program on FDA clinical hold

Sarepta offers early look at closely watched gene therapy launch

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