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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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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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

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

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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Gene Therapy Gene Trials Clinical Trials 246

XTalks

JANUARY 8, 2024

Whether it’s for a treatment for a chronic ambulatory condition, precision medicine or cell and gene therapy, there is a massive uptick in clinical trial complexity. The key considerations she shared include: Project management: The expertise and approach of the team managing the trial.

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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

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

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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BioPharma Reporter

JANUARY 18, 2024

The Cell and Gene Therapy Catapult (CGT Catapult), an independent organization specialising in the advancement of cell and gene therapies, has revealed that the UK has remained an attractive destination for commercial trials, with this type of clinical trial accounting for 81% of trials in 2023 and 80% in 2022.

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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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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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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

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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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

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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Gene Therapy Gene Clinical Trials Clinical Trials 260

BioPharma Reporter

APRIL 4, 2022

The first patient has been dosed in the Phase 1/2 clinical trial of OCU400, a modifier gene therapy candidate for the treatment of retinitis pigmentosa (RP) resulting from mutations in the nuclear receptor subfamily 2 group E member 3 (NR2E3) and Rhodopsin (RHO) genes.

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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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BioPharma Reporter

MARCH 5, 2024

Neurogene, a clinical-stage company developing genetic medicines, is expanding its ongoing phase 1/2 clinical trial investigating NGN-401 as a treatment for female pediatric patients with Rett Syndrome.

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XTalks

JANUARY 17, 2024

Estimates based on publicly available information suggest more than 40 percent of all new therapies in development are cancer treatments. Given this hotbed of activity, innovation in the space to drive faster decisions and more efficient trials is intense.

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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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XTalks

JUNE 14, 2023

Courtney Silverthorn who is an Associate Vice President at the Foundation for the National Institutes of Health (FNIH) and the Director of the Accelerating Medicines Partnership (AMP) program including the Bespoke Gene Therapy Consortium. Subscribe to the Xtalks Life Science Podcast to never miss a new episode.

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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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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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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.

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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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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.

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BioSpace

NOVEMBER 15, 2023

The Japanese pharma contends that an analysis of the four deaths in its AT132 gene therapy clinical trial shows it is still viable as a potential treatment for a fatal, rare genetic disease.

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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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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.

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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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Drug Discovery World

OCTOBER 9, 2023

Positive Phase I trial data indicates that Reqorsa (quaratusugene ozeplasmid) has shown early efficacy for the treatment of non-small cell lung cancer (NSCLC). Developed by gene therapy company Genprex, Reqorsa is a non-viral gene therapy that leads to expression of the TUSC2 tumour suppressor gene in cancers.

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Pharmaceutical Technology

MAY 25, 2023

Pushing back an initial deadline, the US Food and Drug Administration (FDA) has proposed a new regulatory action date of 22 June, by which time the agency will assess the logistics of a possible approval for Sarepta Therapeutics’ Duchenne muscular dystrophy (DMD) gene therapy.

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Drug Discovery World

AUGUST 23, 2022

A Phase I/II clinical trial targeting a treatment for Huntington’s Disease (HD) using a gene therapy has been given the clearance to go ahead in France. . The post Bayer gets go-ahead for Huntington’s Disease gene therapy trial appeared first on Drug Discovery World (DDW).

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Drug Discovery World

OCTOBER 31, 2023

UK regulatory authorities have approved the first trial of a gene therapy for young children with Hunter syndrome. The drug was developed over eight years by Brian Bigger, Professor of Cell and Gene Therapy at The University of Manchester.

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Pharmaceutical Technology

JUNE 19, 2023

Although only a small number of gene therapies have reached the market thus far, the industry is poised to grow quickly over the next few years. According to GlobalData’s clinical trials database, there are currently 1,231 planned and ongoing trials for gene therapies and gene-modified cell therapies alone.

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BioPharma Reporter

DECEMBER 8, 2022

Emmes has announced the creation of a dedicated center for cell and gene therapy research: which will focus on supporting clinical trials for the Clinical Research Organizationâs (CRO) clients.

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