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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Gene Therapy Gene Trials Clinical Trials 95

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.

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Gene Therapy Clinical Trials Clinical Trials Gene 52

XTalks

JANUARY 17, 2024

Changes in what clinical performance data is used to inform the trial design and site selection, new approaches to patient recruitment and the rise of advanced treatments such as cell and gene therapy are rewriting the script. Vidal shared their insights from the 2023 ESMO conference which occurred in Madrid.

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Clinical Trials Clinical Trials Trials Gene Therapy 124

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 85

Gene Therapy Gene Manufacturing Clinical Trials 85

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.

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Gene Therapy Gene Packaging Packaging 155

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.

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Gene Therapy Gene Trials Protein 83

Worldwide Clinical Trials

JANUARY 24, 2024

Much of what we discuss I covered in my presentation, “Leveraging Genetics to Support Rare Disease Clinical Trials,” at last year’s World Orphan Drug Congress (WODC) EU. In my capstone, I focused on how, if, and when genetic counselors discuss clinical trials with their patients in the context of a counseling session.

Genetics 168

Genetics Trials Clinical Trials Clinical Trials 168

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.

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Gene Therapy Gene Production Clinical Trials 52

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).

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Gene Therapy Gene Clinical Trials Clinical Trials 52

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. It has been estimated that by 2025 the FDA will be approving 10 to 20 cell and gene therapy products per year 2.

Gene Therapy 52

Gene Therapy Gene Development Production 52

Drug Discovery World

SEPTEMBER 6, 2022

In recent years the advanced therapy industry has been the most significant disruptor in medicine. Cell and gene therapies are not only transforming the way diseases are treated but are also aiming to cure entirely new patient populations. ViaCyte, Beam, and Verve are all due to begin gene editing trials this year.

Gene Therapy 52

Gene Therapy Gene Gene Editing Manufacturing 52

Drug Discovery World

MAY 16, 2023

What are the global innovations in cell and gene therapy? What opportunities and challenges are emerging and can therapies get to market faster? According to Foster, the industry needs to work together to address the problems from the earliest phases of pre-clinical development. DDW’s Megan Thomas finds out.

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Gene Therapy Gene Manufacturing Marketing 52

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. In a recent webinar, Dr. Vassallo discussed the operational considerations for complex cell therapy clinical trials.

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Clinical Trials Clinical Trials Trials Gene Therapy 52

Drug Discovery World

SEPTEMBER 20, 2022

This will probably include data from the ongoing long-term, follow-up study LTF-304, which follows patients treated in clinical trials for 15 years, and from commercially treated patients. “As The post FDA approves gene therapy for rare neurological disorder appeared first on Drug Discovery World (DDW).

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Gene Therapy FDA Approval Gene Clinical Trials 52

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

Drug Discovery World

SEPTEMBER 12, 2022

Chaired by Karin Blumer, Director of Global Patient Engagement at Novartis, the first event in this track, called ‘Patient Engagement: Driving Patient Advocacy to Inform Patient Centric Development’, looked into the multi-layered importance of involving patients in the drug development process, particularly in this case for gene therapy.

Clinical Trials 52

Clinical Trials Clinical Trials Trials Gene Therapy 52

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.

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In-Vivo Gene Therapy Gene RNA 246

Pharmaceutical Technology

MAY 18, 2023

Forbion European Acquisition has entered a definitive business combination agreement with biotechnology company enGene to create a combined biotechnology company to develop next-generation non-viral, locally administered gene therapies. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Gene Therapy 130

Gene Therapy Gene In-Vivo Life Science 130

Worldwide Clinical Trials

MAY 20, 2022

The past few years we’ve had exciting opportunities to work on more cell and gene therapy programs for our sponsors. The industry is just at the tip of the iceberg for exploring the potential of these therapies in revolutionizing how we treat disease, and as the approvals start to trickle in, the pipeline only grows.

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Gene Therapy Gene Development Research 130

XTalks

MAY 20, 2021

Today is Clinical Trials Day 2021 — a day for recognizing the importance of clinical research in protecting and improving global health. Amid the alarmist headlines posted by other media outlets, Xtalks has remained balanced in its reporting of COVID-19 clinical trial results and safety data.

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Clinical Trials Clinical Trials Trials Vaccination 105

The Pharma Data

JANUARY 17, 2021

LONDON–( BUSINESS WIRE )– Ixaka Ltd , an integrated cell and gene therapy company focused on the natural power of the body to cure disease, launches today. The new business will continue to develop Ixaka’s proprietary technologies – concentrated multi-cell therapies (MCTs) and targeted nanoparticle (TNP) therapeutics.

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Gene Therapy Gene In-Vivo Genetics 40

Drug Discovery World

AUGUST 30, 2022

More than 80 speakers will be giving talks across four content tracks on critical issues that the gene therapy sector is facing. Emily Whitehead became the first child in the world to receive the revolutionary cell therapy treatment named CAR-T in 2012 as a last hope to cure her of leukaemia – she remains in remission 10 years later.

Gene Therapy 98

Gene Therapy Gene Clinical Trials Clinical Trials 98

Roots Analysis

FEBRUARY 27, 2024

Currently, the most popular viral vectors, on the basis of their use in active clinical trials, are those based on AAV, adenovirus, lentivirus and retrovirus. On the other hand, among non-viral gene delivery tools, plasmid DNA has emerged as the preferred option. Overall, eleven gene therapies have been approved so far.

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Manufacturing Gene Therapy Gene DNA 52

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.

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Gene Therapy Gene Trials Clinical Trials 264

Roots Analysis

FEBRUARY 28, 2023

Given the potential of gene therapies in targeting the underlying cause of a disease at cellular level, the demand for such therapies has increased considerably over the past few years. Currently, more than 285 gene therapies are being evaluated in different phases of clinical development.

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Gene Therapy Gene Clinical Trials Clinical Trials 52

Pharmaceutical Technology

AUGUST 17, 2022

Biopharmaceutical contract development and manufacturing organisation (CDMO) AGC Biologics has entered a strategic collaboration with RoosterBio to expedite the manufacturing of cell and exosome therapies. It will also have the potential to scale to Phase III clinical development and commercial manufacturing.

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Manufacturing Gene Therapy Genetic Engineering Gene 130

Advarra

FEBRUARY 24, 2023

One area receiving increased focus from the Food and Drug Administration (FDA) are trial designs incorporating multiple endpoints to support efficacy. Developing the endpoint design and overall approach of a clinical trial program is mostly science, but there is a bit of art to it as well.

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Clinical Trials Clinical Trials Trials Regulation 52

Pharmaceutical Technology

APRIL 5, 2023

But while companies continue studying allogeneic CAR-T therapies, including for their coveted use in solid tumours, such advancements remain challenging. More broadly however, several advancements are on the horizon for cell and gene therapies in 2023. AZ: Cell and gene therapies often come with a high price.

Gene Therapy 147

Gene Therapy Gene Engineer Production 147

Pharmaceutical Technology

FEBRUARY 17, 2023

Capricor will handle the clinical development of CAP-1002 and sell commercial products to Nippon Shinyaku. Additionally, Capricor will receive up to $89m in further development and sales-based milestones, along with a double-digit share of revenue based on product sales.

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Gene Therapy Sales Clinical Development Gene 130

Pharmaceutical Technology

FEBRUARY 8, 2023

Genenta CEO Pierluigi Paracchi said: “The expansion of our existing relationship with AGC Biologics underscores the progress of our platform’s clinical development. Based on results from Phase I of the ongoing Phase I/II clinical trial, we are scaling up the manufacturing process to be ready for Phase II of the trial.

Manufacturing 130

Manufacturing Gene Therapy Production Clinical Trials 130

Delveinsight

JULY 28, 2020

Daiichi’s DS-1062 , a trophoblast cell-surface antigen 2 (TROP2) directed DXd antibody-drug conjugate (ADC) is currently in Phase I clinical trial for multiple cancer types including the majority of non-small cell lung cancer (NSCLC) and triple-negative breast cancer (TNBC). Daiichi holds exclusive rights to the drug in Japan.

Gene Therapy 52

Gene Therapy Trials Gene Vaccination 52

Pharmaceutical Technology

AUGUST 4, 2022

P-BCMA-ALLO1 is currently being analysed in a Phase I clinical trial while an investigational new drug (IND) application for P-CD19CD20-ALLO1 is anticipated next year. Furthermore, Roche will oversee the late-stage clinical development and worldwide marketing of all products under the alliance.

Development 147

Development Clinical Trials Clinical Trials Gene Therapy 147

XTalks

OCTOBER 7, 2020

Not only did this baby survive to do all these things, but he became a poster child for gene therapy with the regulators at the U.S. Born with spinal muscular atrophy (SMA), the little boy was saved by the drug Zolgensma, a one-time intravenous infusion that restored the child’s defective SMN1 gene. Collaboration.

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Development Gene Therapy Clinical Trials Clinical Trials 98

The Pharma Data

AUGUST 7, 2020

The governing body claims this proposal could have positive effects on mitigating burdensome requirements for gene therapy clinical trials in the future. In this light, Pharma IQ’s weekly round-up focuses on advancing therapies and clinical trials to combat Covid-19.

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Clinical Trials Clinical Trials Gene Trials 52

Pharmaceutical Technology

NOVEMBER 21, 2022

Additionally, the wide-ranging expertise of RVAC in mRNA, vaccine and drug delivery technologies, and its capabilities in research and development (R&D), biopharmaceutical manufacturing, clinical development and marketing is leveraged under the partnership.

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Manufacturing Medicine Vaccination Vaccine 130

Pharmaceutical Technology

OCTOBER 28, 2022

Within China, JW Therapeutics will oversee the development, production and marketing of the therapy. The company could also use the initial clinical trial findings generated under the partnership to back the development in various other regions.

Development 130

Development Gene Therapy Production Clinical Trials 130

Drug Discovery World

FEBRUARY 12, 2024

We’re already seeing biotechs making breakthroughs with AI in this area, with more than 150 small molecule drugs discovered and 15 already in clinical trials. Matt Todd, Head of Architecture, Ori Biotech “We expect AI and data analytics to support pivotal advances in cell and gene therapy (CGT) manufacturing.

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Genome Genomics Life Science Gene Therapy 139