Gene Therapy and Regulation - Clinical Research Informer

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.

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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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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.

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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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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.

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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.

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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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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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Cell, gene therapy makers lose a champion at FDA with exit of Peter Marks

Bio Pharma Dive

MARCH 31, 2025

Marks’ resignation leaves the field without a regulator many view as “integral” to its progress over the last decade.

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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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uniQure signs license deal for Apic Bio’s gene therapy for SOD1-ALS

Pharmaceutical Technology

JANUARY 31, 2023

Gene therapy company uniQure has entered into a global licensing agreement with Apic Bio for APB-102 to treat patients with amyotrophic lateral sclerosis (ALS) caused by mutations in superoxide dismutase 1 (SOD1). APB-102 is designed to be a one-time, intrathecally administered gene therapy for ALS patients.

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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 suggests ways to curb constraints with rare disease gene therapy trials

Pharmaceutical Technology

FEBRUARY 8, 2023

On February 7, at a town hall organised to discuss clinical trial designs for gene therapies, FDA experts pushed pharma players to look for ways to establish clinical effectiveness despite the challenges in recruiting patients with rare diseases.

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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.

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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.

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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.

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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.

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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.

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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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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.

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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.

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Bluebird says gene therapy 'very unlikely' cause of cancer case in trial

Bio Pharma Dive

MARCH 10, 2021

and European regulators in hopes of resuming study of the treatment as well as sales of a related product. The biotech is discussing its findings with U.S.

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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.

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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.

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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.

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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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5 FDA decisions to watch in the third quarter

Bio Pharma Dive

JUNE 29, 2022

Between July and September, the regulator could approve a first-of-its-kind autoimmune drug and two gene therapies as well as decide how COVID-19 vaccines should be updated.

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FDA proposes cell and gene therapy site tours to help regulators learn from industry

Fierce Pharma

JULY 15, 2024

It's no secret that cell and gene therapies have faced manufacturing hurdles as the advanced medicines have become increasingly popular in recent years. | It's no secret that cell and gene therapies have faced manufacturing hurdles as the advanced medicines have become increasingly popular in recent years.

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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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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.

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Pfizer scores FDA nod for hemophilia B gene therapy, will charge $3.5M per dose

Fierce Pharma

APRIL 26, 2024

Ten years after dipping its toes into the gene therapy pool in a licensing deal with Spark Therapeutics, Pfizer has gained FDA approval for the acquired treatment. | Ten years after dipping its toe into the gene therapy pool in a licensing deal with Spark Therapeutics, Pfizer has gained an FDA approval for the acquired treatment.

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FDA puts Voyager study on hold in latest gene therapy speed bump

Bio Pharma Dive

OCTOBER 13, 2020

The biotech won't be able to start human testing of an experimental Huntington's disease treatment until regulators see more manufacturing data.

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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.

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Logistics in Focus: Orchestrating the Challenges of Cell Therapy

Worldwide Clinical Trials

NOVEMBER 12, 2024

By Amy Raymond, PhD, PMP, Executive Director, Therapeutic Strategy Lead, Rare Disease Cell and gene therapies (CGTs) include cutting-edge approaches that offer the hope of a healthier, happier, and better tomorrow for a wide range of patient populations. Below, we discuss some of these challenges in cell therapy trials.

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Untangling the Complexities of Cell and Gene Therapy Clinical Trials: A Supply Chain Perspective

Pharmaceutical Technology

MAY 24, 2023

By Luisa Sterkel & Joana Loureiro , Tenthpin Consultants The promise and potential of cell and gene therapies (CGT) has emerged in the recent past and currently over 1.500 CGT are registered for clinical trials holding great hope for the treatment of challenging and uncurable diseases.

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FDA INTERACT Meetings: Early Interactions for Cell and Gene Therapy Sponsors

Camargo

MAY 2, 2021

Advances in scientific knowledge and growth in the cell and gene therapy space have led to a new and exciting era of medicine for patients, as well as a new motivation for regulators to provide clear, efficient pathways for product developers. Background: The Advancement of Cell and Gene Therapies.

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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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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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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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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.

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Astellas makes another gene therapy play, takes stake in Taysha

pharmaphorum

OCTOBER 25, 2022

Astellas has been having some safety-related issues with its gene therapy programmes of late, but it shows no signs of lessened enthusiasm for the category – as a new alliance with US biotech Taysha demonstrates. ” The post Astellas makes another gene therapy play, takes stake in Taysha appeared first on.

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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.

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BioMarin Gene Therapy Halted by FDA Over Safety Concerns

XTalks

SEPTEMBER 9, 2021

The US Food and Drug Administration (FDA) has placed a clinical hold on BioMarin Pharmaceutical’s investigational gene therapy BMN 307 for the rare inherited disease phenylketonuria (PKU) over safety concerns found during preclinical testing. The mice developed the tumors one year after being given BMN 307.

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Novartis adds gene therapy for blindness with Gyroscope buy

pharmaphorum

DECEMBER 22, 2021

Gyroscope Therapeutics and its gene therapy for the sight-robbing disease geographic atrophy (GA) has been acquired by a big pharma company – but likely not the one you would expect. The one-shot therapy aims to treat GA by delivering the gene for complement factor I (CFI) to the eye using an adeno-associated virus (AAV) vector.

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Bluebird's next gene therapy gets backing from European regulator

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

Webinars

Trending Sources

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

Webinars

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

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

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

European regulators back hemophilia gene therapy

Astellas to support development of Taysha’s gene therapy programmes

Cell, gene therapy makers lose a champion at FDA with exit of Peter Marks

Bluebird gene therapy approved by FDA for rare blood disease

uniQure signs license deal for Apic Bio’s gene therapy for SOD1-ALS

Magazine: CRISPR gene therapies cut through in 2023

FDA suggests ways to curb constraints with rare disease gene therapy trials

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

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

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

FDA lifts hold on Astellas gene therapy for Pompe disease

FDA starts clock on review of CSL, UniQure gene therapy

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

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

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

Bluebird says gene therapy 'very unlikely' cause of cancer case in trial

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

Paper details Astellas gene therapy study that led to patient deaths

French biotech pulls EMA application for eye disease gene therapy

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

5 FDA decisions to watch in the third quarter

FDA proposes cell and gene therapy site tours to help regulators learn from industry

PDUFA VII: Implications for Developers of Cell and Gene Therapies

BioMarin reports cancer case in hemophilia gene therapy trial

Pfizer scores FDA nod for hemophilia B gene therapy, will charge $3.5M per dose

FDA puts Voyager study on hold in latest gene therapy speed bump

Gene therapy specialist bluebird exits “untenable” European market

Logistics in Focus: Orchestrating the Challenges of Cell Therapy

Untangling the Complexities of Cell and Gene Therapy Clinical Trials: A Supply Chain Perspective

FDA INTERACT Meetings: Early Interactions for Cell and Gene Therapy Sponsors

Rocket Pharmaceuticals’ gene therapy receives FDA RMAT designation

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

Astellas reports fourth death in halted gene therapy trial

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

Astellas makes another gene therapy play, takes stake in Taysha

Bayer unit BrainVectis cleared to start Huntington gene therapy trial

BioMarin Gene Therapy Halted by FDA Over Safety Concerns

Novartis adds gene therapy for blindness with Gyroscope buy

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