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.

Gene Therapy 296

Gene Therapy Gene Regulation Trials 296

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).

Gene Therapy 264

Gene Therapy Gene Trials Marketing 264

Pharmaceutical Technology

AUGUST 22, 2023

The gene therapy trials in geographic atrophy and Stargardt disease are planned to start by the end of 2023.

Gene Therapy 130

Gene Therapy Gene Trials 130

AuroBlog - Aurous Healthcare Clinical Trials blog

APRIL 29, 2024

The mutation is in the gene for a protein called programmed death-ligand 1 (PD-L1), and a […] A unique genetic mutation in two siblings – that has never been seen in anyone else – has been discovered by UK researchers at the University of Exeter, pointing the way towards new treatment options for type 1 diabetes.

Gene 183

Gene Genetics Protein Research 183

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.

Gene Therapy 246

Gene Therapy Gene DNA Reagent 246

Worldwide Clinical Trials

MARCH 20, 2024

One way to ensure customized disease management is to utilize pharmacogenomics (PGx) in your clinical trial. As the study of how an individual’s genes affect their response to drugs, pharmacogenomics is a critical factor in developing effective and safe drugs and dosages in clinical trials.

Clinical Trials 130

Clinical Trials Clinical Trials Trials Genetics 130

Bio Pharma Dive

MAY 17, 2022

An unusual collaboration among gene therapy developers suggests certain mutations could be behind "peculiar" side effects experienced by several patients treated in clinical trials.

Gene Therapy 339

Gene Therapy Gene Scientist Clinical Trials 339

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.

Gene Therapy 246

Gene Therapy Gene Trials Clinical Trials 246

pharmaphorum

FEBRUARY 5, 2024

4D Molecular Therapeutics' 4D-150 gene therapy for wet AMD has shown efficacy and a reduced need for eye injections with current therapy Eylea in a phase 2 trial.

Gene Therapy 100

Gene Therapy Gene Trials 100

Worldwide Clinical Trials

NOVEMBER 27, 2023

Casgevy, the commercial product formerly known as exa-cel, is administered by taking stem cells out of a patient’s bone marrow and editing a gene in the cells in a laboratory, with the modified cells then infused back into the patient after conditioning treatment to prepare the bone marrow.

Gene Editing 190

Gene Editing Gene Gene Therapy In-Vivo 190

Bio Pharma Dive

JULY 22, 2021

Three companies are testing their gene therapies in early clinical trials, with initial results due later this year. Others are in preclinical stages and aim to follow soon.

Gene Therapy 343

Gene Therapy Gene Clinical Trials Clinical Trials 343

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.

Gene Therapy 246

Gene Therapy Gene Clinical Trials Clinical Trials 246

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.

Gene Therapy 244

Gene Therapy Gene Development Clinical Trials 244

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

Gene Therapy 114

Gene Therapy Clinical Trials Clinical Trials Gene 114

Worldwide Clinical Trials

OCTOBER 24, 2023

Global Genes is a rare disease umbrella organization dedicated to eliminating the burdens and challenges of rare diseases for patients and families globally. Over the years, I have attended several Global Genes Summits. Roughly 80% of rare diseases are due to a known genetic driver.

Gene 130

Gene Gene Editing Gene Therapy Clinical Trials 130

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.

Gene Therapy 264

Gene Therapy Gene Development Genetics 264

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.

Gene Therapy 341

Gene Therapy Trials Gene 341

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.

Gene Therapy 246

Gene Therapy Gene Pharma Companies Clinical Trials 246

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.

Gene Therapy 264

Gene Therapy Gene Clinical Trials Clinical Trials 264

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.

Gene Therapy 272

Gene Therapy Gene Clinical Trials Clinical Trials 272

BioPharma Reporter

OCTOBER 31, 2023

Sarepta Therapeutics has hit a setback as its approved gene therapy Elevidys missed the primary goal of a phase 3 trial in children aged 4 through 7 years with the inherited disease Duchenne muscular dystrophy (DMD).

Gene Therapy 96

Gene Therapy Gene Trials 96

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.

Gene Therapy 98

Gene Therapy Clinical Trials Clinical Trials Gene 98

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.

Gene Therapy 227

Gene Therapy Gene Research Clinical Trials 227

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.

Gene Therapy 326

Gene Therapy Clinical Trials Clinical Trials Trials 326

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.

Gene Therapy 264

Gene Therapy Gene Clinical Trials Clinical Trials 264

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

Clinical Trials 130

Clinical Trials Clinical Trials Gene Marketing 130

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.

Gene Therapy 264

Gene Therapy Manufacturing Gene Clinical Trials 264

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.

Gene Therapy 130

Gene Therapy Gene Clinical Trials Clinical Trials 130

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.

Genetics 190

Genetics Trials Clinical Trials Clinical Trials 190

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 200

Gene Therapy Trials Gene Regulation 200

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.

Gene Therapy 315

Gene Therapy Gene Trials Research 315

pharmaphorum

MAY 7, 2024

Two-year data from a phase 1/2 trial of eyeDNA Therapeutics' gene therapy for retinitis pigmentosa sets up pivotal trials, says the new biotech.

Trials 98

Trials Gene Therapy Gene 98

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.

Gene Therapy 305

Gene Therapy Gene Trials 305

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 95

Gene Therapy Gene Trials Clinical Trials 95

pharmaphorum

SEPTEMBER 26, 2022

Pfizer and Sangamo have restarted recruitment of patients into their registration trial for haemophilia A gene therapy giroctocogene fitelparvovec, after the FDA lifted a clinical hold on the study. The post Pfizer, Sangamo resume haemophilia gene therapy trial appeared first on.

Gene Therapy 98

Gene Therapy Gene Trials Development 98

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.

Gene Therapy 238

Gene Therapy Gene Gene Editing Genetics 238

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.

Gene Therapy 260

Gene Therapy Gene Trials Compliance 260