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Clinical Trials Gene Trials 
XTalks
APRIL 8, 2025
Global rare disease clinical trials face unique challenges due to small patient populations, complex logistics, diverse regulatory environments and cultural differences. Clinical Trial Manager/Sr. These measures enhance recruitment, retention and overall trial success.
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
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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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.
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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.
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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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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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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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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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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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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. As of 2023, there were approximately 2.7
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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
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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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
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.
246 
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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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
NOVEMBER 28, 2022
The challenges biopharma companies face in running clinical trials and tips on how to surmount those obstacles.
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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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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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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
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
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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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.
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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
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.
264 
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.
246 
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.
246 
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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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.
246 
AuroBlog - Aurous Healthcare Clinical Trials blog
SEPTEMBER 22, 2024
(Richard Jones/Science Photo Library/Getty Images) Scientists have discovered hundreds of genes that could potentially promote cancer, new research shows. Cancer is typically triggered by some kind of change to our genetic coding that interferes with a cell’s ability to manage its growth.
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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
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
JUNE 25, 2024
Family members with just a single copy of her version of a particular gene – what’s known as the Christchurch variant of apolipoprotein E (APOE) – seem to also be protected against genetically determined early-onset dementia, researchers […]
245 
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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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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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.
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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. Cold chain logistics, meanwhile, have never been so important.
245 
Worldwide Clinical Trials
DECEMBER 12, 2022
Genetic testing provides patients with a diagnosis for their illness, helps patients and family members to understand risks of developing new diseases, and can be used to support clinical trial advancement. Can prior genetic testing results be utilized in a trial, or should a new genetic sample be taken before participating?
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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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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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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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