Gene 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.

Gene Therapy

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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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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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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.

Gene Therapy

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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

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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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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.

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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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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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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Vertex, CRISPR strengthen case for pioneering gene-editing treatment

Bio Pharma Dive

JUNE 11, 2022

Positive data from 75 patients with either sickle cell disease or beta thalassemia keep the companies’ therapy on track to become the first CRISPR-based treatment submitted to drug regulators, possibly by the end of the year.

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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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FDA sets decision dates for Vertex, CRISPR gene editing drug

Bio Pharma Dive

JUNE 9, 2023

The regulator will issue separate verdicts on the pioneering treatment’s use in sickle cell disease and beta thalassemia, with the first expected late this year.

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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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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

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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 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

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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

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In first, Verve gets clearance to test base editing inside the body

Bio Pharma Dive

MAY 11, 2022

Regulators in New Zealand have allowed human testing to proceed, another milestone for the gene editing technology. Verve plans to soon ask for approval to begin testing in the U.S.

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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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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

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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

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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.

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.

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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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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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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

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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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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.

Gene Therapy

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Sanofi invests in MeiraGTx and its gene-regulating technology

Bio Pharma Dive

OCTOBER 30, 2023

In addition to Sanofi’s investment, the biotech signaled it’s weighing deals with “multiple” other companies that have expressed interest in its work.

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Researchers speed identification of DNA regions that regulate gene expression

Scienmag

MAY 6, 2021

Jude Children’s Research Hospital scientists have developed a highly efficient method to address a major challenge in biology–identifying the genetic ‘switches’ that regulate gene expression. Credit: St. Jude Children’s Research Hospital St.

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European regulators push Biogen, CRISPR drugs toward approval

Bio Pharma Dive

DECEMBER 15, 2023

While a positive recommendation for the gene editing treatment Casgevy was expected, a clearance for Biogen’s Skyclaris, acquired through a recent acquisition, was no sure bet.

Gene Editing

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Gene editing: beyond the hype

pharmaphorum

JANUARY 29, 2021

Cutting edge’ is, for once, a truly apt description when it comes to gene editing – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome. Zinc fingers.

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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. The global nature of cell and gene therapy trials adds another layer of complexity.

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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

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Alyftrek: Vertex Expands Cystic Fibrosis Treatment with Triple-Action Combo

XTalks

JANUARY 6, 2025

The US Food and Drug Administration (FDA) has approved Alyftrek (vanzacaftor/tezacaftor/deutivacaftor), a next-in-class triple combination cystic fibrosis transmembrane conductance regulator (CFTR) modulator, to treat cystic fibrosis (CF) in patients aged six years and older with at least one F508del mutation or another responsive CFTR mutation.

Gene Therapy

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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.

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. This blog post addresses those aspects of PDUFA VII that are expected to affect sponsors of cell and gene therapy products, as well as therapies designed to treat rare diseases.

Gene Therapy

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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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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.

Gene Therapy

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Avance Clinical Expands Gene Technology Clinical Trial Services to Meet $17.4 billion Market Demand

Pharma Mirror

MAY 7, 2021

The leading Australian CRO for biotechs and Frost & Sullivan Asia-Pacific CRO Market Leadership Award winner, Avance Clinical, has expanded its gene technology clinical trial services to meet the increasing global demand predicted to reach $17.4 The post Avance Clinical Expands Gene Technology Clinical Trial Services to Meet $17.4

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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.

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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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Bluebird's next gene therapy gets backing from European regulator

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

Webinars

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

Webinars

European regulators back hemophilia gene therapy

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

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

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

Bluebird gene therapy approved by FDA for rare blood disease

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

Astellas to support development of Taysha’s gene therapy programmes

Vertex, CRISPR strengthen case for pioneering gene-editing treatment

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

FDA sets decision dates for Vertex, CRISPR gene editing drug

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

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

Magazine: CRISPR gene therapies cut through in 2023

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

FDA lifts hold on Astellas gene therapy for Pompe disease

In first, Verve gets clearance to test base editing inside the body

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

FDA starts clock on review of CSL, UniQure gene therapy

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

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

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

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

Paper details Astellas gene therapy study that led to patient deaths

French biotech pulls EMA application for eye disease gene therapy

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

5 FDA decisions to watch in the third quarter

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

Sanofi invests in MeiraGTx and its gene-regulating technology

Researchers speed identification of DNA regions that regulate gene expression

European regulators push Biogen, CRISPR drugs toward approval

Gene editing: beyond the hype

Logistics in Focus: Orchestrating the Challenges of Cell Therapy

BioMarin reports cancer case in hemophilia gene therapy trial

Alyftrek: Vertex Expands Cystic Fibrosis Treatment with Triple-Action Combo

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

PDUFA VII: Implications for Developers of Cell and Gene Therapies

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

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

Avance Clinical Expands Gene Technology Clinical Trial Services to Meet $17.4 billion Market Demand

Three regulators accept Bristol Myers’ applications for myeloma therapy

Gene therapy specialist bluebird exits “untenable” European market

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