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

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

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.

Gene Therapy 264

Gene Therapy Gene Trials Clinical Trials 264

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.

Gene Therapy 264

Gene Therapy Gene Clinical Trials Clinical Trials 264

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.

Clinical Trials 246

Clinical Trials Clinical Trials Gene Therapy Clinical Supply Chain 246

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

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.

Gene Therapy 264

Gene Therapy Manufacturing Gene Clinical Trials 264

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

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

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

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

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

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.

Gene Therapy 245

Gene Therapy Gene Medicine Genetics 245

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.

Gene Therapy 244

Gene Therapy Gene Genetics Clinical Research 244

Bio Pharma Dive

NOVEMBER 28, 2022

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

Gene Therapy 288

Gene Therapy Gene Clinical Trials Clinical Trials 288

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.

Clinical Trials 287

Clinical Trials Clinical Trials Branding Gene Therapy 287

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

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.

Clinical Trials 280

Clinical Trials Clinical Trials Gene Therapy Gene 280

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

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.

Gene Therapy 305

Gene Therapy Gene Clinical Trials Clinical Trials 305

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.

Gene Therapy 265

Gene Therapy Gene Marketing Clinical Trials 265

Pharmaceutical Technology

JULY 22, 2022

Pipeline therapies within the diabetic macular oedema (DME) space have recently gathered interest following the American Society of Retina Specialists (ASRS) Annual Meeting, which took place on 13–16 July.

Gene Therapy 260

Gene Therapy Gene Trials Compliance 260

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. This can be a devastating outcome when trying again just isn’t possible.

Gene Therapy 182

Gene Therapy Clinical Supply Trials Gene 182

Bio Pharma Dive

JUNE 7, 2021

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

Gene Therapy 245

Gene Therapy Gene Clinical Trials Clinical Trials 245

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.

Gene Therapy 260

Gene Therapy Gene Clinical Trials Clinical Trials 260

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.

Gene Therapy 171

Gene Therapy Gene Clinical Trials Clinical Trials 171

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.

Gene Therapy 246

Gene Therapy Clinical Trials Clinical Trials Gene 246

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

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.

Gene Therapy 184

Gene Therapy Gene Development Production 184

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 122

Gene Therapy Clinical Trials Clinical Trials Gene 122

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

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.

Gene Therapy 105

Gene Therapy Clinical Trials Clinical Trials Gene 105

Pharmaceutical Technology

JULY 20, 2022

Avista Therapeutics, a University of Pittsburgh Medical Center (UPMC) spinout, has entered a collaboration with Roche for developing new AAV gene therapy vectors for eyes. Roche will use these new capsids to carry out preclinical, clinical and marketing activities for gene therapy programmes.

Gene Therapy 147

Gene Therapy Gene Development In-Vivo 147

Pharmaceutical Technology

JULY 14, 2022

Avrobio has received orphan drug designation for its gene therapy, AVR-RD-05, from the US Food and Drug Administration (FDA) to treat mucopolysaccharidosis type II (MPSII) or Hunter syndrome. The company noted that this gene therapy is the fourth one to receive orphan drug designation.

Gene Therapy 130

Gene Therapy Gene Drugs In-Vivo 130

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.

Trials 104

Trials Clinical Trials Clinical Trials Gene Therapy 104

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.

Gene Therapy 100

Gene Therapy Clinical Trials Clinical Trials Gene 100

Worldwide Clinical Trials

JUNE 15, 2022

At the end of May, we hosted a webinar titled “ Changing Times, Changing Therapies: Keeping Up with Advancements in Cell and Gene Therapies ” to provide a quick update on the latest advancements and ongoing in development of these advanced therapeutics. Around 40% of clinical holds are for gene therapy programs.

Gene Therapy 130

Gene Therapy Gene Clinical Trials Clinical Trials 130