Gene Therapy and Regulation - Clinical Research Informer

PTC's first gene therapy gains European regulator's backing

Bio Pharma Dive

MAY 20, 2022

The brain-infused treatment for a rare neurological disease would be one of only three gene therapies commercially available in the EU. PTC plans to ask for FDA approval later this year.

Gene Therapy

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Bluebird's next gene therapy gets backing from European regulator

Bio Pharma Dive

MAY 21, 2021

The treatment, for a progressive, often deadly brain disease, could soon become Bluebird's third approved product and one of only a handful of marketed gene therapies in the world.

Gene Therapy

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ASGCT2024: FDA’s Marks on Accelerated Approval, Gene Therapy Costs and More

BioSpace

MAY 8, 2024

In a fireside chat at the American Society of Gene & Cell Therapy conference, CBER Director Peter Marks spoke with Takeda’s Kristin Van Goor about how the regulator is approaching the exploding gene therapy space.

Gene Therapy

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US gene therapy approvals create a big problem for Brazil. Regulators have to come together, FDA's Peter Marks says

Fierce Pharma

MAY 9, 2024

US gene therapy approvals create a big problem for Brazil. Regulators have to come together, FDA's Peter Marks says aarmstrong Thu, 05/09/2024 - 10:17

Gene Therapy

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European regulators back hemophilia gene therapy

Bio Pharma Dive

DECEMBER 16, 2022

The EMA’s drug review committee recommended five new drugs be approved in Europe during its December meeting, including CSL’s Hemgenix, AstraZeneca’s Imjudo and Amicus’ Pombiliti.

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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.

Gene Therapy

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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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Another Astellas gene therapy trial paused by FDA after side effect report

Bio Pharma Dive

JUNE 27, 2022

The regulator suspended testing of Astellas’ Pompe disease treatment after one patient experienced peripheral nerve damage. Both of the pharma’s clinical-stage gene therapies are now on hold.

Gene Therapy

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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.

Gene Therapy

Gene Therapy Gene Regulation

Magazine: CRISPR gene therapies cut through in 2023

Pharmaceutical Technology

FEBRUARY 2, 2023

If things go as per plan, in a few months, the US Food and Drug Administration (FDA) will deliberate on the first-of-its-kind CRISPR-based gene therapy for sickle cell disease (SCD) and transfusion-dependent beta thalassemia. The post Magazine: CRISPR gene therapies cut through in 2023 appeared first on Pharmaceutical Technology.

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FDA wants more details on Sarepta gene therapy, potentially delaying final test

Bio Pharma Dive

SEPTEMBER 10, 2020

A new request from the regulator could tighten the race between Sarepta and Pfizer to prove out a gene therapy for Duchenne muscular dystrophy.

Gene Therapy

Gene Therapy Gene Regulation

4DMT’s CF gene therapy posts early cues for improving patient outcomes

Pharmaceutical Technology

JUNE 8, 2023

The use of 4D Molecular Therapeutics’ (4DMT) aerosolised gene therapy 4D-710 has improved the quality-of-life and spirometry-measured outcomes in three cystic fibrosis patients , based on early results from a Phase I/II study presented at this year’s annual meeting of the European Cystic Fibrosis Society (ECFS).

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Gene therapy could reduce seizures in childhood epilepsy

Drug Discovery World

DECEMBER 18, 2023

University College London (UCL) researchers have developed a new gene therapy to cure focal cortical dysplasia, which a new study shows can significantly reduce seizures in mice. They then injected either the virus carrying the EKC gene or a control virus into the affected brain area.

Gene Therapy

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Rocket Pharmaceuticals’ gene therapy receives FDA RMAT designation

Pharmaceutical Technology

MAY 24, 2023

The RMAT designation programme is intended to accelerate the drug’s development and review processes for products, including gene therapies. The regulator granted RMAT designation based on the data obtained from the ongoing Phase I RP-L301 clinical trial.

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Paper details Astellas gene therapy study that led to patient deaths

Bio Pharma Dive

NOVEMBER 16, 2023

The company, which is working with regulators to lift a clinical hold, said the treatment could still help people with X-linked myotubular myopathy.

Gene Therapy

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FDA Accepts Orchard’s BLA for Rare Disease Gene Therapy Behind EU Approval

BioSpace

SEPTEMBER 18, 2023

regulator has accepted Orchard Therapeutics’ BLA for its gene therapy OTL-200, being proposed for metachromatic leukodystrophy. Nearly three years after European approval, the U.S.

Gene Therapy

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Bluebird gene therapy approved by FDA for rare blood disease

Bio Pharma Dive

AUGUST 17, 2022

The regulator cleared the biotech’s medicine Zynteglo for transfusion-dependent beta thalassemia, giving patients a powerful new treatment option. But it will come at a very high cost of $2.8 million in the U.S.

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STAT+: Differences in countries’ regulations keep some cell and gene therapies from reaching patients

STAT News

OCTOBER 12, 2022

— Biotech companies developing cutting-edge cell and gene therapies often are tripped up by how much regulations around these drugs vary from country to country. On Wednesday, regulators from the United States, the European Union, and Japan came together to discuss the scale of the problem — and possible solutions.

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FDA lifts hold on Astellas gene therapy for Pompe disease

Bio Pharma Dive

JANUARY 20, 2023

The regulator’s decision ends a seven-month study pause that followed a report of one participant experiencing mild symptoms of peripheral neuropathy.

Gene Therapy

Gene Therapy Gene Regulation

FDA reversal sets up high-stakes hearing on Sarepta gene therapy

Bio Pharma Dive

MARCH 17, 2023

The regulator will convene a panel of advisers to consult on an approval decision for the biotech’s Duchenne treatment, a change from just a few weeks ago when Sarepta had said a meeting wouldn’t be held.

Gene Therapy

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French biotech pulls EMA application for eye disease gene therapy

Bio Pharma Dive

APRIL 21, 2023

With cash running low and a search for a buyer underway, GenSight wants advice from regulators on the type of data that would support an approval.

Gene Therapy

Gene Therapy Gene Regulation

FDA Approves Two Gene Therapies for Sickle Cell, Including First CRISPR-Based.

BioSpace

DECEMBER 7, 2023

Along with CRISPR/Cas9-based Casgevy—developed by Vertex Pharmaceuticals and CRISPR Therapeutics—the regulator on Friday approved bluebird bio’s Lyfgenia, a second gene therapy for sickle cell disease.

Gene Therapy

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Bluebird, playing catch-up, gets decision date for sickle cell gene therapy

Bio Pharma Dive

JUNE 21, 2023

The regulator will issue a decision on lovo-cel by Dec. 20, roughly two weeks after a verdict is expected on a rival gene editing treatment from Vertex and CRISPR Therapeutics.

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Bluebird’s Lovo-Cel Gene Therapy for SCD Will Not Face FDA Adcomm

BioSpace

AUGUST 15, 2023

The regulator informed bluebird bio that it will not convene an advisory committee meeting to discuss the company’s application for the gene therapy being developed for sickle cell disease.

Gene Therapy

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BioMarin says gene therapy hold will endure as FDA questions cancer risk

Bio Pharma Dive

FEBRUARY 18, 2022

According to BioMarin, the regulator has requested additional data to assess the "theoretical" cancer risk to the treatment, information that will take "several quarters" to collect.

Gene Therapy

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Cell and Gene Therapy Catapult, Rentschler Biopharma and Refeyn establish new gene therapies partnership

BioPharma Reporter

AUGUST 24, 2023

Cell and Gene Therapy Catapult, Rentschler Biopharma, and Refeyn have announced a new partnership to develop and apply âinnovativeâ process analytical technologies (PAT) to improve the process and efficiency of AAV manufacture.

Gene Therapy

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PDUFA VII: Implications for Developers of Cell and Gene Therapies

Camargo

DECEMBER 20, 2021

Many of the advancements included in PDUFA VII are driven in large part by an increase in the development of innovative products such as cell and gene therapies. The increase is designed to strengthen CBER’s capacity and capabilities for regulating cell and gene therapies. 2024: 48 staffers. 2025: 29 staffers.

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FDA starts clock on review of CSL, UniQure gene therapy

Bio Pharma Dive

MAY 25, 2022

The regulator agreed to assess the companies' data under priority review, setting up a decision on approval of the hemophilia B treatment in six months.

Gene Therapy

Gene Therapy Gene Regulation

FDA clears Pfizer to restart Duchenne gene therapy trial, with new safeguards

Bio Pharma Dive

APRIL 28, 2022

The regulator has asked Pfizer to closely monitor patients in a hospital setting for a week as part of a deal to end a study suspension that's lasted more than four months.

Gene Therapy

Gene Therapy Gene Regulation Trials

BioSpace Heads to #ASGCT, Along with Thousands in Cell and Gene Therapy

BioSpace

MARCH 4, 2024

Follow News Editor Greg Slabodkin and Managing Editor Jef Akst as they travel with some 8,000 others for discussions of cell and gene therapy advances, challenges, regulations and more.

Gene Therapy

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SK pharmteco to manufacture Ferring Pharmaceuticals’ gene therapy for bladder cancer

BioPharma Reporter

APRIL 22, 2024

SK pharmteco has signed an agreement with Ferring Pharmaceuticals, to scale up commercial manufacturing capacity for the drug substance of Ferringâs intravesical gene therapy Adstiladrin.

Gene Therapy

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Bayer unit BrainVectis cleared to start Huntington gene therapy trial

pharmaphorum

AUGUST 23, 2022

France’s BrainVectis, a subsidiary of Bayer’s Asklepios BioPharma (AskBio) unit, has been given the green light by regulators in France to start dosing patients with its gene therapy candidate for devastating neurodegenerative disorder Huntington’s disease.

Gene Therapy

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BioMarin reports cancer case in hemophilia gene therapy trial

Bio Pharma Dive

SEPTEMBER 13, 2022

Drug regulators have not ordered a trial hold, however. The development comes three weeks after Roctavian was approved in Europe and ahead of a planned regulatory submission in the U.S.

Gene Therapy

Gene Therapy Trials Gene Regulation

How to advance AAV-based gene therapies

Drug Discovery World

OCTOBER 18, 2023

Sara Donnelly, Director of Research Planning and Business Development at PhoenixBio USA explores why the right pre-clinical model is essential for teams wanting to advance adeno-associated virus vector-based gene therapies.

Gene Therapy

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eBook: Analytical methods for viral vector development in gene therapy

Drug Discovery World

SEPTEMBER 6, 2023

The eleventh eBook of the Hamilton series ‘Analytical Methods for Viral Vector Development and Manufacturing in Gene Therapy’ is now available.

Gene Therapy

Gene Therapy Gene Development Manufacturing

10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases

XTalks

APRIL 24, 2023

Awareness of rare diseases is growing, and with a better understanding of the pathophysiology of many rare diseases, innovative treatment options are emerging, like gene therapies that can treat the root cause of rare genetic diseases and potentially provide long-term symptom relief, or even a definitive cure.

Gene Therapy

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Analytical methods for viral vector development and manufacturing in gene therapy

Drug Discovery World

SEPTEMBER 5, 2023

In the eleventh eBook of the Hamilton series ‘Analytical Methods for Viral Vector Development and Manufacturing in Gene Therapy’, we learn about: Gene therapy: history, viral vectors and the role of CDMOs Automation of analytical assays during viral vector development and manufacturing The solutions that Hamilton offers to customers working (..)

Gene Therapy

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Gene therapy specialist bluebird exits “untenable” European market

pharmaphorum

AUGUST 10, 2021

Europe market has proved so hostile to gene therapies when it comes to pricing and reimbursement for gene therapies that bluebird bio has decided to quit the market altogether, according to Andrew Obenshain, president of its severe genetic diseases unit. — Brad Loncar (@bradloncar) August 9, 2021.

Gene Therapy

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Bluebird, after delays, gets speedy FDA review for beta thalassemia gene therapy

Bio Pharma Dive

NOVEMBER 22, 2021

The regulator will decide whether to approve Bluebird's treatment by next May, but its evaluation begins more than a year later than the biotech first hoped.

Gene Therapy

Gene Therapy Gene Regulation

Can vertical AI advance cell and gene therapies?

Drug Discovery World

DECEMBER 8, 2023

Andrew Busey , Co-Founder, Form Bio discusses why vertical AI is set to reshape the cell and gene therapy sector. The cell and gene therapy industry is no exception to this digital transformation. Here’s what this means for pharmaceutical leaders and patients.

Gene Therapy

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Sarepta, with new Duchenne results, to broach 'possibility' of speedy gene therapy approval

Bio Pharma Dive

JANUARY 10, 2022

regulators, CEO Doug Ingram said Monday, though it still expects to need data from an ongoing late-stage trial to win approval. The biotech will take new Phase 2 data to U.S.

Gene Therapy

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FDA clears Rocket to resume testing gene therapy for a rare heart disease

Bio Pharma Dive

AUGUST 16, 2021

The decision from the regulator ends a three-month study pause that had stalled Rocket's development of of the Danon disease treatment.

Gene Therapy

Gene Therapy Gene Regulation Development

Astellas reports fourth death in halted gene therapy trial

pharmaphorum

SEPTEMBER 14, 2021

There has been a fourth patient fatality in Astellas’ clinical trial of its AT132 gene therapy for the rare disease X-linked myotubular myopathy (XLMTM), which has been halted twice due to safety concerns. The post Astellas reports fourth death in halted gene therapy trial appeared first on.

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Three regulators accept Bristol Myers’ applications for myeloma therapy

Pharmaceutical Technology

APRIL 18, 2023

Three regulators in different jurisdictions have accepted Bristol Myers Squibb’s applications for Abecma (idecabtagene vicleucel) for earlier use to treat triple-class exposed relapsed and/or refractory multiple myeloma in adult patients. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Regulation

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PTC's first gene therapy gains European regulator's backing

Bluebird's next gene therapy gets backing from European regulator

Trending Sources

ASGCT2024: FDA’s Marks on Accelerated Approval, Gene Therapy Costs and More

US gene therapy approvals create a big problem for Brazil. Regulators have to come together, FDA's Peter Marks says

European regulators back hemophilia gene therapy

At FDA meeting, gene therapy experts wrestle with field's blindspots

Astellas to support development of Taysha’s gene therapy programmes

Another Astellas gene therapy trial paused by FDA after side effect report

What to expect at the FDA's two-day meeting on gene therapy safety

Magazine: CRISPR gene therapies cut through in 2023

FDA wants more details on Sarepta gene therapy, potentially delaying final test

4DMT’s CF gene therapy posts early cues for improving patient outcomes

Gene therapy could reduce seizures in childhood epilepsy

Rocket Pharmaceuticals’ gene therapy receives FDA RMAT designation

Paper details Astellas gene therapy study that led to patient deaths

FDA Accepts Orchard’s BLA for Rare Disease Gene Therapy Behind EU Approval

Bluebird gene therapy approved by FDA for rare blood disease

STAT+: Differences in countries’ regulations keep some cell and gene therapies from reaching patients

FDA lifts hold on Astellas gene therapy for Pompe disease

FDA reversal sets up high-stakes hearing on Sarepta gene therapy

French biotech pulls EMA application for eye disease gene therapy

FDA Approves Two Gene Therapies for Sickle Cell, Including First CRISPR-Based.

Bluebird, playing catch-up, gets decision date for sickle cell gene therapy

Bluebird’s Lovo-Cel Gene Therapy for SCD Will Not Face FDA Adcomm

BioMarin says gene therapy hold will endure as FDA questions cancer risk

Cell and Gene Therapy Catapult, Rentschler Biopharma and Refeyn establish new gene therapies partnership

PDUFA VII: Implications for Developers of Cell and Gene Therapies

FDA starts clock on review of CSL, UniQure gene therapy

FDA clears Pfizer to restart Duchenne gene therapy trial, with new safeguards

BioSpace Heads to #ASGCT, Along with Thousands in Cell and Gene Therapy

SK pharmteco to manufacture Ferring Pharmaceuticals’ gene therapy for bladder cancer

Bayer unit BrainVectis cleared to start Huntington gene therapy trial

BioMarin reports cancer case in hemophilia gene therapy trial

How to advance AAV-based gene therapies

eBook: Analytical methods for viral vector development in gene therapy

10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases

Analytical methods for viral vector development and manufacturing in gene therapy

Gene therapy specialist bluebird exits “untenable” European market

Bluebird, after delays, gets speedy FDA review for beta thalassemia gene therapy

Can vertical AI advance cell and gene therapies?

Sarepta, with new Duchenne results, to broach 'possibility' of speedy gene therapy approval

FDA clears Rocket to resume testing gene therapy for a rare heart disease

Astellas reports fourth death in halted gene therapy trial

Three regulators accept Bristol Myers’ applications for myeloma therapy

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