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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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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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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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). The patient suffered acute liver failure several months after receiving the therapy in December. Late in 2024, Elevidys was recognized among Times Best Innovations.

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Bio Pharma Dive

DECEMBER 11, 2023

Editas had the tall task Monday of convincing ASH attendees its gene therapy for sickle cell disease could improve on Casgevy and Lyfgenia.

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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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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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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

JUNE 27, 2023

A Duchenne gene therapy faces a crucial test, while highly anticipated study results are expected in lung cancer, obesity and heart disease.

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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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Bio Pharma Dive

NOVEMBER 28, 2022

The challenges biopharma companies face in running clinical trials and tips on how to surmount those obstacles.

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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. Personalised medicines will also be developed at the centre.

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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

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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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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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

FEBRUARY 16, 2021

The biotech is also suspending sales of its related treatment Zynteglo following a leukemia diagnosis in a clinical trial volunteer and a case of a cancer-like bone marrow disease in another.

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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. In the Phase I trial, 12 participants received the therapy and were observed for two years.

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Bio Pharma Dive

JULY 9, 2021

Zynteglo sales have been on hold since February, when a patient in a clinical trial of another, related Bluebird medicine developed leukemia.

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Pharmaceutical Technology

MAY 3, 2024

The FDA has awarded the designation following a review of initial safety and efficacy data from two Phase I/II clinical trials.

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Pharmaceutical Technology

MAY 30, 2023

Amplo Biotechnology has received a fast track phase I/II STTR grant from the NIH-NIAMS [National Institutes of Health’s National Institute of Arthritis and Musculoskeletal and Skin Diseases] for its gene therapy AMP-201. The company will receive substantial funding to advance AAV-ColQ gene therapy.

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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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Pharmaceutical Technology

JULY 7, 2022

For many decades, investigators have been working on innovative therapeutic modalities known as cell and gene therapies, which use modified versions of the body’s own cellular and genetic material to treat and potentially cure these diseases. A new frontier in cancer research.

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Pharmaceutical Technology

MAY 16, 2023

The Foundation for the National Institutes of Health (FNIH) has announced its plans to prioritise eight rare diseases to provide industry standards for manufacturing, preclinical testing and product analytical testing for gene therapy development. This will include pairing up indications with manufacturers amongst the BGTC’s partners.

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AuroBlog - Aurous Healthcare Clinical Trials blog

SEPTEMBER 9, 2024

The convergence of gene therapies and clinical research is pushing the boundaries of what’s possible in ophthalmic care, offering hope for more effective treatments and potential cures for a range of vision-threatening conditions.

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Bio Pharma Dive

JUNE 7, 2021

Shorter timelines for clinical trials means being ready earlier for later stage trial requirements and regulatory reviews.

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Pharmaceutical Technology

JANUARY 24, 2024

The company could be in line for a priority review voucher if 4D-710, currently in Phase I/II clinical trials, is approved.

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Bio Pharma Dive

MARCH 19, 2021

Stung by a clinical trial miss in Duchenne muscular dystrophy, the biotech now has promising, if early, data to back a gene therapy for a different rare neuromuscular disease.

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Bio Pharma Dive

SEPTEMBER 13, 2021

The CGT industry is expected to grow 30% between 2019 and 2025, and the transition from clinical trials to commercial-scale production is now seen as inevitable.

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Bio Pharma Dive

MAY 16, 2023

Backed by nearly $100 million, the public-private consortium aims to create a standard development roadmap for gene therapies using AAV viral vectors.

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pharmaphorum

FEBRUARY 27, 2024

Looking to the future of clinical trials: Gene therapy, precision medicine, and the ongoing quest for rare disease solutions Mike.Hammerton Tue, 27/02/2024 - 12:30 Bookmark this

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XTalks

MAY 17, 2023

The Foundation for the National Institutes of Health (FNIH) announced this week that the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC) has selected eight rare diseases for its clinical trial portfolio.

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BioPharma Reporter

AUGUST 24, 2022

BrainVectis, a subsidiary of AskBio, has received clearance from the French authorities to start a Phase 1-2 clinical trial for its novel Huntingtonâs Disease gene therapy, BV-101.

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Fierce Pharma

DECEMBER 2, 2024

The FDA is investigating reports of patients develo | The FDA is investigating reports of patients developing blood cancer after receiving bluebird bio’s gene therapy Skysona and is evaluating the need for “further regulatory action,” the U.S. agency said last week.

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Pharma Times

JANUARY 20, 2025

Cell and Gene Therapy Catapult reveals significant growth in UK trials

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Worldwide Clinical Trials

JANUARY 24, 2024

Written By: Derek Ansel, MS, CCRA, Executive Director, Therapeutic Strategy Lead, Rare Disease Given that 80% of rare diseases have a genetic etiology, genetic implications should be addressed at the onset of a clinical program to support trial enrollment. One diagnostic example that I discussed in my presentation is autism.

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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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