Clinical Development, Gene and Trials - Clinical Research Informer

FDA suggests ways to curb constraints with rare disease gene therapy trials

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

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Astellas to support development of Taysha’s gene therapy programmes

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

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EC grants approval for BioMarin’s gene therapy to treat Hemophilia A

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

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Leveraging Genetic Testing for Enrolling Rare Disease Trials

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.

Genetics

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Gene Therapy and Pharmacokinetics

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

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Precision eye therapy for dogs ready for human clinical development

Medical Xpress

APRIL 26, 2023

A successful gene therapy trialed at Michigan State University in dogs with an inherited eye disease is ready to be developed for clinical use in human patients with a rare condition called retinitis pigmentosa.

Clinical Development

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10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases

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.

Gene Therapy

Gene Therapy Gene Trials Clinical Trials

Cell and gene therapies: why advanced medicines call for specialised logistics

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.

Gene Therapy

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?5 Things You Need to Know from Our Recent Gene Therapy Webinar

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. Clinical holds are becoming more common, especially in gene therapy programs.

Gene Therapy

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Verve Therapeutics and Lilly partner to advance gene editing programme

Pharmaceutical Technology

JUNE 15, 2023

Verve Therapeutics and Eli Lilly and Company have entered an exclusive research partnership to advance the former’s preclinical stage in vivo gene editing programme targeting lipoprotein(a) (Lp(a)) to treat atherosclerotic cardiovascular disease (ASCVD). Lilly will provide funding for the Phase I clinical trials.

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First UK patient joins Pfizer’s phase 3 DMD gene therapy trial

pharmaphorum

MAY 13, 2021

The first UK patient has enrolled on Pfizer’s phase 3 trial for its Duchenne muscular dystrophy (DMD) gene therapy. The first patient was recruited in The Newcastle upon Tyne Hospitals NHS Foundation Trust, one of three UK sites for the clinical trial and part of 55 globally, across 15 countries.

Gene Therapy

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UK maintains dominant global position in cell and gene therapy

Drug Discovery World

JANUARY 22, 2024

The UK is cementing its position as a global leader in cell and gene therapy (CGT), according to a new report released today by the UK BioIndustry Association (BIA), in collaboration with Citeline. The UK has 84 drugs in clinical development, surpassing all other European countries, with 23 CGTs are already approved for patient use in the UK.

Gene Therapy

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FDA gives Ferring go-ahead for Adstiladrin gene therapy manufacturing

Pharmaceutical Technology

JUNE 13, 2023

The US Food and Drug Administration (FDA) approved Ferring Pharmaceuticals’ Prior-Approval Supplement (PAS) to the Biologics License Application (BLA) for the bladder cancer gene-therapy Adstiladrin (nadofaragene firadenovec). The therapy received approval based on positive Phase III trial (NCT02773849) results.

Gene Therapy

Gene Therapy Manufacturing Gene FDA Approval

PDUFA VII: Implications for Developers of Cell and Gene Therapies

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. 2026: 15 staffers.

Gene Therapy

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Present Treatments & Hopeful Future Directions for ALS Pharmacotherapies

Worldwide Clinical Trials

MAY 30, 2024

Authors: Rich Worldwide Clinical Trials Exec. This trend is on the rise despite recent disappointments with clinical trial outcomes, which have the potential to destabilize the industry in the short term regarding drug development strategy and optimal study designs. Director, Therapeutic Area Medical Lead.

Gene

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Potential first-in-class therapeutic for urea cycle disorders enters trial

Drug Discovery World

MARCH 26, 2024

Mutations in genes encoding urea cycle enzymes result in insufficient levels of these important proteins. CMP-CPS-001 is an antisense oligonucleotide (ASO) designed to amplify CPS1 mRNA by harnessing fundamental cellular gene expression control mechanisms.

Trials

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Gene Therapy Clinical Trials for Rare Diseases: 5 Key Takeaways from FDA’s Recent Town Hall

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

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What Are the Top Oncology Clinical Trial Trends Through the Lens of ESMO?

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.

Clinical Trials

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Astellas takes $170m charge as it drops DMD gene therapies

pharmaphorum

APRIL 22, 2022

Astellas has said it will book a $170 million impairment charge in its fourth quarter results as a result of a decision to halt the development of three gene therapy candidates for Duchenne muscular dystrophy in preclinical development. The post Astellas takes $170m charge as it drops DMD gene therapies appeared first on.

Gene Therapy

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FDA INTERACT Meetings: Early Interactions for Cell and Gene Therapy Sponsors

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

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Finding a cure: How cell and gene therapies could revolutionise medicine

Pharmaceutical Technology

JANUARY 26, 2023

Cell and gene therapies are predicted to be the future of medical treatment by providing the body with the means to repair itself and recover from a range of serious conditions and severe diseases. While cell and gene therapies are currently much more expensive than traditional medical treatments, the ultimate savings could be significant.

Gene Therapy

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Dyno attracts $1.6bn gene therapy deal with Astellas

pharmaphorum

DECEMBER 2, 2021

Astellas has signed another bolt-on deal to build its gene therapy pipeline, agreeing a deal with Dyno Therapeutics to tap into its adeno-associated virus (AAV) vector platform for delivering genes to skeletal and cardiac muscle. Those safety issues have hit Astellas’ aspirations in gene therapy directly.

Gene Therapy

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FDA grants approval for bluebird’s Zynteglo to treat beta-thalassemia

Pharmaceutical Technology

AUGUST 18, 2022

A custom-made, one-dose gene therapy, Zynteglo is indicated for such patients who need red blood cells (RBCs) transfusions on a regular basis. The latest approval is based on findings from the Phase III clinical trials, HGB-207 (Northstar-2) and HGB-212 (Northstar-3), and the long-term follow-up LTF-303 study.

Gene Therapy

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FDA approves first gene therapy for haemophilia B

Drug Discovery World

NOVEMBER 24, 2022

The US Food and Drug Administration (FDA) has approved HEMGENIX (etranacogene dezaparvovec-drlb), the first and only one-time gene therapy for appropriate adults with haemophilia B. . The FDA approval is supported by results from the ongoing HOPE-B trial, the largest gene therapy trial in haemophilia B to date.

Gene Therapy

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Novartis adds gene therapy for blindness with Gyroscope buy

pharmaphorum

DECEMBER 22, 2021

Gyroscope Therapeutics and its gene therapy for the sight-robbing disease geographic atrophy (GA) has been acquired by a big pharma company – but likely not the one you would expect. The one-shot therapy aims to treat GA by delivering the gene for complement factor I (CFI) to the eye using an adeno-associated virus (AAV) vector.

Gene Therapy

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First gene therapy for haemophilia B approved in Great Britain

Drug Discovery World

MARCH 31, 2023

CSL’s Hemgenix (etranacogene dezaparvovec) has been authorised as the first gene therapy for adults with severe and moderately severe haemophilia B (congenital Factor IX (FIX) deficiency) in Great Britain. The post First gene therapy for haemophilia B approved in Great Britain appeared first on Drug Discovery World (DDW).

Gene Therapy

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Gene-edited cell replacement therapy for diabetes set to enter the clinic

BioPharma Reporter

NOVEMBER 16, 2021

have received the green light from Health Canada for their Clinical Trial Application for VCTX210: an allogeneic, gene-edited, immune-evasive, stem cell-derived therapy for the treatment of type 1 diabetes (T1D). CRISPR Therapeutics and ViaCyte, Inc.

Gene Editing

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Intellia Gets FDA Clearance to Start First Ever Phase III Trial for an In Vivo CRISPR Drug

XTalks

OCTOBER 20, 2023

Clinical-stage genome editing company Intellia Therapeutics has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to start a pivotal phase III trial of NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy.

In-Vivo

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US FDA approves Pfizer’s migraine nasal spray Zavzpret

Pharmaceutical Technology

MARCH 10, 2023

Zavzpret is claimed to be the first and only calcitonin gene-related peptide (CGRP) receptor antagonist nasal spray approved to treat migraine. It is a third-generation, selective and structurally unique, small molecule CGRP receptor antagonist and is currently under clinical development with oral and intranasal formulations.

FDA Approval

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Myeloid raises funds to support lead cell therapy programme

Pharmaceutical Technology

MAY 19, 2023

Myeloid Therapeutics has raised $73m to support the continued clinical development of its lead cell therapy programme, MT-101, in Phase I/II trials for T cell lymphoma. The financing will help to fast-track the development of other in vivo programming candidates into clinical trials.

In-Vivo

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Another Astellas gene therapy hits a safety hurdle

pharmaphorum

JUNE 27, 2022

The safety of Astellas’ gene therapy portfolio has been thrust into the spotlight once again, after the FDA placed a clinical hold on a trial of its Pompe disease candidate AT845. The delivery vector – an AAV8 capsid – is designed to allow gene expression directly in target tissues like skeletal muscle.

Gene Therapy

Gene Therapy Gene Gene Expression Trials

FDA approves Phase 1 trial for HIV gene therapy

The Pharma Data

AUGUST 24, 2020

The FDA has approved a request from American Gene Technologies to begin a clinical study into its HIV gene therapy. The treatment is being researched by scientists collaborating from American Gene Technologies, the Laboratory of Immunoregulation and the National Institute of Allergy and Infectious Diseases.

Gene Therapy

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Regulatory Trends in Cell and Gene Therapies

Advarra

NOVEMBER 29, 2022

The field of cell and gene therapies (CGT) is constantly evolving, and there has been significant progress in this area of research. However, despite the promise of these therapies, the regulations governing them lag the science, which in turn hinders the clinical translation of these novel medicines.

Gene Therapy

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Potential HIV cure Phase I trial gets FDA green light

Outsourcing Pharma

AUGUST 11, 2020

American Gene Technologies has received the agencyâs approval to launch the first human trial for its lead HIV program, starting in Baltimore and DC.

Trials

Trials Gene Clinical Development Development

Frontotemporal dementia: the state of treatment development

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

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EU-approved gene therapy reduced haemophilia bleeds by 64%

Drug Discovery World

FEBRUARY 22, 2023

The multi-year clinical development of Hemgenix was led by uniQure and sponsorship of the clinical trials transitioned to CSL after it licensed global rights to commercialise the treatment. The post EU-approved gene therapy reduced haemophilia bleeds by 64% appeared first on Drug Discovery World (DDW).

Gene Therapy

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First gene therapy for haemophilia B gets go ahead in Europe

Drug Discovery World

DECEMBER 20, 2022

The first gene therapy for haemophilia B could soon be available to patients in Europe following a positive opinion from the Committee for Medicinal Products for Human Use (CHMP). . The post First gene therapy for haemophilia B gets go ahead in Europe appeared first on Drug Discovery World (DDW). World’s most expensive drug.

Gene Therapy

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Scleroderma drug trial shows up to 60% improvement

Drug Discovery World

NOVEMBER 30, 2023

Biotechnology company Certa Therapeutics has revealed positive Phase II clinical trial data for FT011 in patients with scleroderma. It is important to advance safe and effective treatments through clinical development, given the limited therapeutic options for scleroderma patients.

Drug Trials

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Cell and gene therapies: Opportunities for innovation

Drug Discovery World

SEPTEMBER 6, 2022

Cell and gene therapies are not only transforming the way diseases are treated but are also aiming to cure entirely new patient populations. As researchers and developers work to transform the way we treat diseases, cell and gene therapies will continue to be the centre of innovation in the coming years. . billion raised.

Gene Therapy

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Astellas says Pompe gene therapy clears safety hurdle

pharmaphorum

FEBRUARY 8, 2022

With the safety of gene therapies thrown into the spotlight by a series of FDA clinical holds, Astellas has said that an interim readout in a phase 1/2 trial of its Pompe disease candidate AT845 hasn’t found any cause for concern. The post Astellas says Pompe gene therapy clears safety hurdle appeared first on.

Gene Therapy

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Cell and gene therapy – where is the innovation?

Drug Discovery World

SEPTEMBER 16, 2022

W here globally is innovation occurring and what factors – industry, regulatory, and academia – are helping developers get their products to market? Here, DDW Editor Reece Armstrong explores the global hubs of innovation for cell and gene therapies. The UK stands as one of the key markets for cell and gene therapies.

Gene Therapy

Gene Therapy Gene Manufacturing Medicine

Strategies to Successfully Manage Complex Cell Therapy Clinical Trials

XTalks

FEBRUARY 10, 2023

According to a recent review published in Nature , as of April 2022 almost 1,800 active cell therapy clinical trials were listed in ClinicalTrials.gov, a 33 percent increase from 2021. Among 20 recent trials, 17 experienced delays in meeting key milestones.

Clinical Trials

Clinical Trials Clinical Trials Trials Gene Therapy

Transcending expectations for cell & gene therapy development

Drug Discovery World

JULY 11, 2022

David Lewandowski, Business Director of Cell & Gene Therapy at Azenta Life Sciences, explains how efficient sample management can help to work efficiently and bring therapies to market faster. The global cell and gene therapy (CGT) market reached a value of ~$4 billion in 2020 and is expected to grow to ~$34 billion by 2030 1.

Gene Therapy

Gene Therapy Gene Development Production

FibroGen and Fortis sign exclusive licence deal for mCRPC drug candidate

Pharmaceutical Technology

MAY 9, 2023

FibroGen has signed an exclusive licence agreement with Fortis Therapeutics for the FOR46 antibody-drug conjugate (ADC) that targets a new epitope on CD46, a protein-coding gene. FOR46 is currently being investigated in a Phase I trial as a potential treatment for metastatic castration-resistant prostate cancer (mCRPC).

Drugs

Drugs Clinical Development Antibody Protein

FDA suggests ways to curb constraints with rare disease gene therapy trials

Astellas to support development of Taysha’s gene therapy programmes

Trending Sources

EC grants approval for BioMarin’s gene therapy to treat Hemophilia A

Leveraging Genetic Testing for Enrolling Rare Disease Trials

Gene Therapy and Pharmacokinetics

Precision eye therapy for dogs ready for human clinical development

10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases

Cell and gene therapies: why advanced medicines call for specialised logistics

?5 Things You Need to Know from Our Recent Gene Therapy Webinar

Verve Therapeutics and Lilly partner to advance gene editing programme

First UK patient joins Pfizer’s phase 3 DMD gene therapy trial

UK maintains dominant global position in cell and gene therapy

FDA gives Ferring go-ahead for Adstiladrin gene therapy manufacturing

PDUFA VII: Implications for Developers of Cell and Gene Therapies

Present Treatments & Hopeful Future Directions for ALS Pharmacotherapies

Potential first-in-class therapeutic for urea cycle disorders enters trial

Gene Therapy Clinical Trials for Rare Diseases: 5 Key Takeaways from FDA’s Recent Town Hall

What Are the Top Oncology Clinical Trial Trends Through the Lens of ESMO?

Astellas takes $170m charge as it drops DMD gene therapies

FDA INTERACT Meetings: Early Interactions for Cell and Gene Therapy Sponsors

Finding a cure: How cell and gene therapies could revolutionise medicine

Dyno attracts $1.6bn gene therapy deal with Astellas

FDA grants approval for bluebird’s Zynteglo to treat beta-thalassemia

FDA approves first gene therapy for haemophilia B

Novartis adds gene therapy for blindness with Gyroscope buy

First gene therapy for haemophilia B approved in Great Britain

Gene-edited cell replacement therapy for diabetes set to enter the clinic

Intellia Gets FDA Clearance to Start First Ever Phase III Trial for an In Vivo CRISPR Drug

US FDA approves Pfizer’s migraine nasal spray Zavzpret

Myeloid raises funds to support lead cell therapy programme

Another Astellas gene therapy hits a safety hurdle

FDA approves Phase 1 trial for HIV gene therapy

Regulatory Trends in Cell and Gene Therapies

Potential HIV cure Phase I trial gets FDA green light

Frontotemporal dementia: the state of treatment development

EU-approved gene therapy reduced haemophilia bleeds by 64%

First gene therapy for haemophilia B gets go ahead in Europe

Scleroderma drug trial shows up to 60% improvement

Cell and gene therapies: Opportunities for innovation

Astellas says Pompe gene therapy clears safety hurdle

Cell and gene therapy – where is the innovation?

Strategies to Successfully Manage Complex Cell Therapy Clinical Trials

Transcending expectations for cell & gene therapy development

FibroGen and Fortis sign exclusive licence deal for mCRPC drug candidate

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