XTalks

APRIL 8, 2025

There are over 10,000 rare diseases affecting an estimated 300 million people worldwide where 80% are genetic , 95% lack approved treatments and nearly half begin in childhood. Clinical Trial Manager/Sr. These measures enhance recruitment, retention and overall trial success.

Clinical Trials 88

Clinical Trials Clinical Trials Trials Gene Therapy 88

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.

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Gene Therapy Clinical Supply Trials Gene 182

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.

Genetics 189

Genetics Trials Clinical Trials Clinical Trials 189

Pharma Mirror

APRIL 28, 2021

Catalent will provide process development and CGMP manufacturing of AavantiBio’s adeno-associated viral (AAV) vector-based therapeutic candidate for use in clinical trials in the U.S. and Europe. Catalent will further support process optimization and look to reduce material.

Gene Therapy 130

Gene Therapy Genetic Disease Genetics Gene 130

Pharmaceutical Technology

MAY 3, 2023

The US Food and Drug Administration (FDA) has granted Tenaya Therapeutics’ gene therapy TN-201 a fast track designation for the treatment of myosin binding protein C3 (MYBPC3)-associated hypertrophic cardiomyopathy (HCM). If successful, TN-201 would be a first-in-class disease-modifying gene therapy for MYBPC3-associated HCM.

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Gene Therapy Gene Protein Trials 242

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

AUGUST 18, 2022

The US Food and Drug Administration (FDA) has granted approval for bluebird bio ’s Zynteglo (betibeglogene autotemcel, beti-cel) for the treatment of the underlying genetic cause of beta?thalassemia A custom-made, one-dose gene therapy, Zynteglo is indicated for such patients who need red blood cells (RBCs) transfusions on a regular basis.

Gene Therapy 246

Gene Therapy Gene Genotype In-Vivo 246

XTalks

JANUARY 6, 2025

CF is a progressive genetic disease caused by defective CFTR proteins, which are crucial for regulating salt and water movement in cells. In clinical trials, Alyftrek demonstrated non-inferiority to Trikafta, showing similar improvements in lung function and a reduction in sweat chloride levels.

Gene Therapy 105

Gene Therapy Protein Genetic Disease Gene 105

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.

Gene Therapy 130

Gene Therapy Gene Clinical Trials Clinical Trials 130

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.

Gene Therapy 130

Gene Therapy Gene Licensing Licensing 130

Camargo

MAY 2, 2021

Advances in scientific knowledge and growth in the cell and gene therapy space have led to a new and exciting era of medicine for patients, as well as a new motivation for regulators to provide clear, efficient pathways for product developers. Background: The Advancement of Cell and Gene Therapies.

Gene Therapy 155

Gene Therapy Gene Packaging Packaging 155

AuroBlog - Aurous Healthcare Clinical Trials blog

SEPTEMBER 30, 2024

The development of innovative therapeutic approaches in healthcare encompass a variety of fields, including gene therapy, personalized medicine, immunotherapy, and advanced biologics. The current research areas of interest in India include precision medicine which treatments based on individual genetic profiles to improve […]

Medicine 189

Medicine Research Gene Therapy Genetics 189

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.

Gene Therapy 130

Gene Therapy Gene Packaging Packaging 130

pharmaphorum

JUNE 15, 2021

When Biogen acquired Nightstar Therapeutics in 2019, it added a pair inherited retinal disorder (IRD) gene therapies that it hoped would accelerate a push into the fast-evolving category. . The post Biogen’s ambitions in gene therapy hit by another failed pivotal trial appeared first on.

Gene Therapy 105

Gene Therapy Gene Trials Genetics 105

pharmaphorum

MAY 18, 2022

SwanBio Therapeutics has raised $56 million in a second-round financing that will be used to advance its lead gene therapy candidate for adrenomyeloneuropathy (AMN) into clinical testing later this year. The therapy is designed to compensate for the disease-causing ABCD1 gene.

Gene Therapy 98

Gene Therapy Gene Trials Clinical Trials 98

pharmaphorum

FEBRUARY 11, 2022

Data from the first patients enrolled into Regenxbio’s trial of its gene therapy for rare inherited disease mucopolysaccharidosis type I (MPS I) – also known as Hurler syndrome – has shown the first signs of clinical activity.

Gene Therapy 111

Gene Therapy Gene Trials FDA Approval 111

Worldwide Clinical Trials

NOVEMBER 27, 2023

In recognition of the unmet need and medical urgency for innovative therapies in the sickle cell space, the FDA granted exa-cel Priority Review, with a formal decision expected by December 8, 2023. Why is Casgevy a Breakthrough Gene Therapy for Patients with Sickle Cell Disease?

Gene Editing 189

Gene Editing Gene Gene Therapy In-Vivo 189

pharmaphorum

MAY 21, 2021

US biotech bluebird bio has had a challenging time in the last few months, so a recommendation for EU approval of its gene therapy for adrenoleukodystrophy (ALD) will give it a lift. After two years’ follow-up, 90% of the boys given the gene therapy showed minimal loss of function and were still alive.

Gene Therapy 96

Gene Therapy Gene FDA Approval Clinical Trials 96

pharmaphorum

AUGUST 1, 2022

Sarepta is pressing forward with a bold plan to file with the FDA for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy (DMD) in the next few months, with a view to making it available in sometime around the middle of 2023.

Gene Therapy 105

Gene Therapy Gene Genetics Trials 105

pharmaphorum

JANUARY 3, 2023

As December 2022 closed out, Pfizer announced positive top-line results from its phase 3 BENEGENE-2 study evaluating fidanacogene elaparvovec (SPK-9001), its investigational gene therapy for treatment of adult males with moderately severe to severe haemophilia B.

Gene Therapy 98

Gene Therapy Gene Medicine Trials 98

Pharmaceutical Technology

JUNE 29, 2022

The European Commission (EC) has granted marketing authorisation for Gilead Sciences’ subsidiary Kite’s CAR T-cell therapy, Yescarta (axicabtagene ciloleucel), to treat adults with relapsed or refractory follicular lymphoma (FL). . Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Gene Therapy Clinical Trials Clinical Trials Genetics 278

pharmaphorum

SEPTEMBER 6, 2021

BioMarin Pharma has had another setback in its gene therapy development programme, announcing this morning that the FDA has placed a phase 1/2 trial of its candidate for phenylketonuria (PKU) on hold while it investigates a safety signal. The post FDA slaps clinical hold on BioMarin’s PKU gene therapy appeared first on.

Gene Therapy 94

Gene Therapy Gene Genetic Disease DNA 94

Pharmaceutical Technology

DECEMBER 12, 2022

The alliance will utilise the capabilities of both firms, including international cell therapy leadership and manufacturing of Kite. An investigational late-stage product candidate, CART-ddBCMA is being analysed in the Phase II iMMagine-1 clinical trial at present. By Cytiva Thematic.

Development 264

Development Gene Therapy Clinical Trials Clinical Trials 264

Pharmaceutical Technology

NOVEMBER 7, 2022

A lead product candidate of the company, SNK01 is presently being analysed as a single agent and along with other agents, including checkpoint inhibitors and cell engagers, in trials to treat advanced refractory solid tumours. . Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Gene Therapy 246

Gene Therapy Clinical Trials Clinical Trials Trials 246

Pharmaceutical Technology

FEBRUARY 20, 2023

Different approaches that are studied include antisense oligonucleotides (ASOs), and gene therapies, which are in early clinical trials. Other lines of research look at the genetic overlap between FTD and amyotrophic lateral sclerosis (ALS), which could be used in the development of treatments for both conditions.

Development 246

Development Gene Therapy Protein Gene 246

Medical Xpress

MAY 12, 2023

While not every form of blindness can be cured, recent scientific breakthroughs have uncovered new ways to treat some forms of inherited blindness through gene therapy. Around 43 million of those people are living with blindness.

Gene Therapy 85

Gene Therapy Gene Clinical Trials Clinical Trials 85

Pharmaceutical Technology

JULY 29, 2022

CAMP4’s CSO David Bumcrot PhD tells Pharmaceutical Technology that the company plans to see clinical trials go forward for their urea cycle disorder programs late next year. However, in patients with urea cycle disorders, genetic defects result in inadequate amounts of the enzymes needed to convert nitrogen into urea.

RNA 245

RNA Gene Expression Gene Gene Therapy 245

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.

Clinical Trials 59

Clinical Trials Clinical Trials Gene Therapy Trials 59

XTalks

NOVEMBER 6, 2020

Canadian clinical-stage biotech company Symvivo Corporation has developed an oral COVID-19 vaccine that entered clinical trials this week. The first healthy volunteer was dosed with the vaccine in Australia as part of the bacTRL-Spike COVID-19 Phase I clinical trial. BacTRL Gene Therapy Platform.

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Clinical Trials Clinical Trials Vaccination Vaccine 75

BioPharma Reporter

APRIL 7, 2021

The UK has a strong genetics research base: but to date, academics have found it difficult to progress gene therapy research into clinical trials and beyond. The Sheffield Gene Therapy Innovation and Manufacturing Centre (GTIMC) is one of three new hubs, announced this month, that will tackle these challenges.

Gene Therapy 63

Gene Therapy Gene Manufacturing Clinical Trials 63

Pharmaceutical Technology

SEPTEMBER 22, 2022

Avrobio has received rare pediatric disease designation from the US Food and Drug Administration (FDA) for its investigational gene therapy, AVR-RD-04, designed to treat cystinosis. AVR-RD-04 works by genetically modify a patient's hematopoietic stem cells (HSCs) to express the gene that encodes the cystinosin protein.

Gene Therapy 147

Gene Therapy Gene Protein Clinical Trials 147

XTalks

MARCH 13, 2023

The US Food and Drug Administration’s (FDA) Office of Tissues and Advanced Therapies (OTAT) held a recent town hall where three experts from the regulator provided guidance on how to design and conduct gene therapy clinical trials for rare diseases. It’s a very exciting time in gene therapy.

Gene Therapy 52

Gene Therapy Gene Clinical Trials Clinical Trials 52

Pharmaceutical Technology

MAY 22, 2023

Vyjuvek is a non-invasive, topical, re-dosable gene therapy that delivers functional human COL7A1 gene copies to offer wound healing. The regulatory approval was supported by data from the GEM-1/2 and GEM-3 clinical trials. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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FDA Approval Gene Therapy Genetic Disease Gene 130

pharmaphorum

FEBRUARY 23, 2022

As investment in gene therapies continues to grow rapidly, more effectively engaging patients throughout development has become a priority for any company serious about building safer and more meaningful gene therapy programs. The post 2nd Annual Gene Therapy Patient Engagement Summit appeared first on.

Gene Therapy 52

Gene Therapy Gene Gene Editing Clinical Trials 52

STAT News

APRIL 3, 2023

When he enrolled in a clinical trial a few years ago, the teenager didn’t know which wound on his body was treated with the experimental medication, and which was dosed with a placebo. This trial wasn’t like so many others where some enrollees get the drug in question and others get a sham dose.

Gene Therapy 119

Gene Therapy Gene Clinical Trials Clinical Trials 119

Pharmaceutical Technology

APRIL 6, 2023

Ginkgo Bioworks has strengthened its end-to-end R&D capabilities in gene therapy with the purchase of adeno-associated virus (AAV) capsid discovery and engineering assets from StrideBio, for an undisclosed sum. The new capabilities and IP will be incorporated under Ginkgo’s end-to-end AAV gene therapy development platform.

Engineer 130

Engineer Gene Therapy Gene Manufacturing 130

Scienmag

MAY 11, 2021

An international team of researchers at Great Ormond Street Hospital (GOSH), and University of California, Los Angeles (UCLA) have developed a gene therapy that successfully treated 48 out of 50 children with a form of severe combined immunodeficiency that leaves them without an immune system.

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Gene Therapy Gene Development Research 73