Bio Pharma Dive

NOVEMBER 5, 2020

Sarepta and Pfizer are advancing their experimental treatments into late-stage trials, while others are accelerating research in what's become one of gene therapy's most active fields.

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Bio Pharma Dive

SEPTEMBER 28, 2021

Outside trial data reviewers have attributed three cases of severe muscle weakness to Pfizer’s treatment, causing the company to change the design of its closely watched Phase 3 study.

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Bio Pharma Dive

JUNE 27, 2022

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

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Bio Pharma Dive

OCTOBER 4, 2021

Initiation of the Phase 3 trial is an important milestone for the biotech after earlier setbacks, as well as for patients with the inherited muscle disease.

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Bio Pharma Dive

JANUARY 7, 2021

The first patient enrolled in a Phase 3 trial of Pfizer's treatment was given the experimental one-time therapy on Dec.

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XTalks

NOVEMBER 14, 2024

PTC Therapeutics has gained US Food and Drug Administration (FDA) approval for its new gene therapy, Kebilidi (eladocagene exuparvovec), for treating aromatic L-amino acid decarboxylase (AADC) deficiency. Kebilidi has a known safety profile from clinical trials. for this purpose.

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Bio Pharma Dive

JULY 23, 2021

Five trial participants with diabetic macular edema who received Adverum Biotechnologies' treatment experienced side effects never before reported for an eye gene therapy.

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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.

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Bio Pharma Dive

APRIL 3, 2023

Cell & gene therapy clinical research may help treat diseases and change how we approach healthcare.

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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. million, contributing to a full-year revenue of $820.8

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Bio Pharma Dive

MAY 3, 2022

While a voluntary pause in dosing new patients will remain in place, the FDA's decision puts the study back on track to deliver data in 2023.

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Bio Pharma Dive

SEPTEMBER 14, 2021

The study, which was suspended following three deaths last year, had been restarted in February after Astellas lowered the treatment dose used.

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XTalks

JANUARY 16, 2025

BlueRock Therapeutics, a subsidiary of Bayer AG, is making waves in the treatment of Parkinsons disease by advancing its investigational cell therapy, bemdaneprocel, to a Phase III clinical trial. The clinical advancement follows a thorough review of Phase I trial data under the FDAs Regenerative Medicine Advanced Therapy (RMAT) designation.

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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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Bio Pharma Dive

FEBRUARY 10, 2022

Safety concerns, including the recent death of a patient in an early study, led the FDA to place a hold on the therapy and Pfizer to redesign its Phase 3 trial.

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Bio Pharma Dive

JULY 7, 2022

A year after reporting side effects “not seen before in ocular gene therapy” in a clinical trial, the biotech is eliminating 38% of its workforce to save cash while it runs a new study.

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Bio Pharma Dive

JUNE 21, 2021

The next six months could feature clinical milestones for CRISPR gene editing, the treatment of COVID-19, microbiome drugs and gene-targeted cancer therapy.

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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. Operationalizing these trials requires proactive and flawless management at every stage.

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Bio Pharma Dive

MAY 7, 2024

The company is working with trial researchers to investigate further. Pfizer said the patient, a young boy who was treated earlier last year, had died suddenly.

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Pharmaceutical Technology

NOVEMBER 23, 2022

The US Food and Drug Administration (FDA) has granted approval for CSL Behring’s adeno-associated virus vector-based gene therapy, Hemgenix (etranacogene dezaparvovec), to treat haemophilia B (congenital Factor IX deficiency) in adult patients. The trial is designed to analyse the safety and efficacy of Hemgenix.

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Bio Pharma Dive

APRIL 4, 2022

Safety issues in clinical trials cast doubt on the ability of gene therapy to safely mitigate disease, prompting further research into understanding the virus-host interaction that resulted in new generations of viral vectors.

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Pharmaceutical Technology

SEPTEMBER 15, 2023

The company is also planning to expand its Phase I/II PRODYGY lead candidate trial in retinitis pigmentosa to three more US sites.

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Bio Pharma Dive

JUNE 13, 2024

The failure of another trial adds to uncertainty around the benefits of the gene-based treatments, though industry analysts still expect Sarepta’s Elevidys’ to be unaffected.

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Pharmaceutical Technology

AUGUST 25, 2022

The European Commission (EC) has granted conditional marketing authorisation (CMA) for BioMarin Pharmaceutical ’s gene therapy, Roctavian (valoctocogene roxaparvovec), to treat adults with severe haemophilia A (congenital Factor VIII deficiency). Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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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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Pharmaceutical Technology

APRIL 3, 2023

The deal will see Polyplus join the German life science group’s portfolio allowing the latter to leverage expertise in transfection reagents and plasmid DNA for gene therapy. Polyplus, based in Strasbourg, France, produces key components in the production of viral vectors used in cell and gene therapies.

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Camargo

JULY 27, 2021

How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Gene therapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19. Gene Therapy Definition.

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Bio Pharma Dive

SEPTEMBER 2, 2022

Preliminary results from a Phase 1 study show Sangamo’s treatment to be safe and suggest it is working as intended, leading the biotech to move into the trial’s next phase.

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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. The asset, now transferred to Beacon Therapeutics, is currently in Phase II clinical trials.

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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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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. We also explore how AI is being used to design digital twins for clinical trials.

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Pharmaceutical Technology

JULY 22, 2022

The spotlight was placed on many up-and-coming pharmacotherapies for retinal diseases, one of which was AbbVie’s/Regenxbio’s gene therapy RGX-314. Clinical trial results in DR patients have shown that 47% of eyes treated with RGX-314 have experienced diabetic retinopathy severity score (DRSS) improvements of two or more steps.

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Bio Pharma Dive

AUGUST 3, 2021

After reviewing animal study data submitted by the Swiss pharma, the FDA cleared spinal injections of Zolgensma for study in older patients with SMA.

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Bio Pharma Dive

MAY 27, 2021

Safety monitors cleared the company to test a higher dose after reviewing data from 10 patients, providing some hopeful news after recent setbacks for experimental drugs targeting the genetic disease.

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Pharmaceutical Technology

MARCH 10, 2023

On 10 March, the National Health Service Blood and Transplant (NHSBT) opened a new Clinical Biotechnology Centre (CBC) with the aim of improving the UK’s ability to develop and manufacture cell and gene therapies. In the past, researchers have often had to seek help from outside the UK, thus delaying clinical trials and patient access.

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Bio Pharma Dive

APRIL 3, 2025

The Swiss drugmaker, which holds European rights to Elevidys, suspended three trials while researchers investigate the death of a young man who died following treatment.

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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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