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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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

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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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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Pharmaceutical Technology

AUGUST 22, 2023

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

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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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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.

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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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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

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.

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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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Pharmaceutical Technology

SEPTEMBER 15, 2023

The company is also planning to expand its Phase I/II PRODYGY lead candidate trial in retinitis pigmentosa to three more US sites.

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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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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.

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Pharmaceutical Technology

JANUARY 5, 2023

Capsida Biotherapeutics and Eli Lilly and Company ’s wholly owned subsidiary Prevail Therapeutics have announced a partnership for the development of non-invasive gene therapies for central nervous system (CNS) diseases. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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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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Pharmaceutical Technology

JUNE 8, 2023

The use of 4D Molecular Therapeutics’ (4DMT) aerosolised gene therapy 4D-710 has improved the quality-of-life and spirometry-measured outcomes in three cystic fibrosis patients , based on early results from a Phase I/II study presented at this year’s annual meeting of the European Cystic Fibrosis Society (ECFS).

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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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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.

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Gene Therapy Gene Trials 312

Fierce Pharma

APRIL 23, 2024

Dating to a 2019 clinical hold of a phase 3 trial, getting its gene therapy ra | Dating to a 2019 clinical hold, getting its gene therapy pz-cel across the FDA finish line has been a challenge for troubled Abeona Therapeutics. Now the wait continues as the FDA has rejected the treatment due to CMC issues.

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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).

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BioSpace

MARCH 26, 2024

Nanoscope Therapeutics nabbed a victory on the changed primary endpoint of its Phase IIb vision loss gene therapy trial Tuesday, teeing the biotech up to file for FDA approval of the candidate this year.

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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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Pharmaceutical Technology

FEBRUARY 28, 2024

AGTC-501 is in Phase II development for retinitis in the SKYLINE trial (NCT03316560) and addresses unmet needs within the retinitis space

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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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pharmaphorum

JANUARY 23, 2024

Eli Lilly’s gene therapy for otoferlin-related deafness has restored the hearing of the first child enrolled in its ongoing phase 1/2 trial

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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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Fierce Pharma

JANUARY 3, 2024

Gene therapies have been approved for certain patients with beta thalassemia. Gene therapies have been approved for certain patients with beta thalassemia. After a successful phase 3 readout, Agios hopes it can reach a unique subgroup and eventually all patients.

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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.

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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.

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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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Drug Discovery World

APRIL 19, 2024

Askbio’s AB-1005 (also known as AAV2-GDNF), an investigational gene therapy for the treatment of Parkinson’s disease, was well tolerated with no serious adverse events in a Phase Ib clinical trial. mL) were well tolerated, with no SAEs associated with the investigational gene therapy or contrast agent.

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Pharmaceutical Technology

MAY 12, 2023

Editas Medicine will release new efficacy and safety results of its gene therapy EDIT-301 in severe sickle disease as part of an oral presentation at the European Hematology Association’s (EHA) Hybrid Congress. In December 2022, the company presented data from the first two patients treated in the Phase I/II RUBY (NCT04853576) trial.

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Pharmaceutical Technology

MAY 24, 2023

The RMAT designation programme is intended to accelerate the drug’s development and review processes for products, including gene therapies. The regulator granted RMAT designation based on the data obtained from the ongoing Phase I RP-L301 clinical trial.

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Drug Discovery World

DECEMBER 18, 2023

University College London (UCL) researchers have developed a new gene therapy to cure focal cortical dysplasia, which a new study shows can significantly reduce seizures in mice. The researchers plan to conduct a first-in-human clinical trial in the next five years.

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pharmaphorum

JANUARY 12, 2024

Bayer’s AskBio gene therapy unit has started recruiting patients into a phase 2 trial of its AB-1002 candidate for congestive heart failure.

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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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