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

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 Clinical Trials Clinical Trials Gene 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.

Gene Therapy 52

Gene Therapy Gene Manufacturing Marketing 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

Drug Discovery World

FEBRUARY 12, 2024

In the upcoming year, we will see the expansion of new, more complex markets and updated requirements from regulators. Ensuring sufficient and timely patient enrollment in a rapidly growing number of cell and gene therapy clinical trials is becoming a bottleneck to advancing potentially life-saving therapies.

Genome 139

Genome Genomics Life Science Gene Therapy 139

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 264

Gene Therapy Gene Trials Clinical Trials 264

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.

Development 98

Development Gene Therapy Clinical Trials Clinical Trials 98

XTalks

MARCH 28, 2024

The FDA’s approval of Duvyzat for DMD, based on our robust and successful clinical development program, reflects Italfarmaco’s commitment to providing a safe and proven-effective therapy that can have a meaningful impact for people living with DMD,” said Paolo Bettica, MD, PhD, chief medical officer at Italfarmaco Group in a press release.

FDA Approval 105

FDA Approval Genetics Gene Therapy Gene 105

pharmaphorum

JULY 20, 2021

Bayer subsidiary BlueRock Therapeutics has been granted a fast-track review by the FDA for DA01, its stem cell-based therapy for Parkinson’s disease which is currently in early-stage clinical testing. The post FDA gives speedy review to Bayer’s Parkinson’s stem cell therapy appeared first on.

Gene Therapy 98

Gene Therapy Regulation Gene Trials 98

XTalks

APRIL 12, 2021

Like other neurodegenerative disorders, such as Alzheimer’s disease and dementias, Parkinson’s disease research has been immensely challenging for biotechs, with a high rate of late-stage attrition in trials leading to a lack of disease-modifying therapies being approved. Axovant Gene Therapies. Prevail Therapeutics.

Gene Therapy 96

Gene Therapy Gene Trials Development 96

FDA Law Blog

NOVEMBER 2, 2023

While on the surface this may not seem like major advance for the field, this could be a game changer for the gene therapy space. The standards would, as stated previously, be voluntary, unless mandated by statute or regulation, and they cannot conflict with existing law or regulation.

Medicine 59

Medicine Gene Therapy Gene Production 59

XTalks

FEBRUARY 10, 2023

Jeff Vassallo, Senior Director of Clinical Trial Management, at the global clinical contract research organization (CRO) Medpace. These details must be thoughtfully considered early in clinical development to ensure studies are completed within the projected timeline and budget,” added Dr. Vassallo.

Clinical Trials 52

Clinical Trials Clinical Trials Trials Gene Therapy 52

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

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. It’s very positive and it’s going to help move forward the pathway for new drugs.”

Clinical Trials 124

Clinical Trials Clinical Trials Trials Gene Therapy 124

Camargo

JULY 30, 2021

In the Pediatric Study Plan, you have a defined timeline and a defined plan, a clinical development plan that has to be approved by the FDA. That has to do a lot with the work in gene therapy and rare disease and the ultra-rare space because there are 7,000 rare diseases that have been identified.

Clinical Research 226

Clinical Research Research Gene Therapy Gene 226

Drug Discovery World

JUNE 15, 2023

The International Society for Cellular Therapy (ISCT) is a global society of clinicians, regulators, technologists, and industry partners with a shared mission to drive the translation of all cell and gene (CGT) therapies for the benefit of patients worldwide. Here, attendees weigh in on future regulatory changes.

Regulation 52

Regulation Production Development Medicine 52

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 52

Gene Therapy Gene Manufacturing Medicine 52

pharmaphorum

AUGUST 12, 2020

The FDA has started its review of Israeli biotech Protalix BioTherapeutics and partner Chiesi’s Fabry disease therapy pegunigalsidase alfa, setting up a possible approval by 27 January. billion by 2027, driven by new oral drugs such as Galafold as well as gene therapies that are still in clinical development.

Drugs 95

Drugs Genetic Disease Gene Therapy Trials 95

Drug Discovery World

MAY 6, 2024

A more established Amsterdam-based enterprise is gene therapy company uniQure, developer of etranacogene dezaparvovec, the first gene therapy for haemophilia B, which was approved for use in the US in November 2022 and in the EU in February 2023. The CDD will sponsor and manage KWF-supported trials.

Drugs 59

Drugs Life Science Gene Editing Gene 59

FDA Law Blog

JANUARY 4, 2023

The purpose of these town halls are to discuss topics related to OTAT-regulated products, engage with product development stakeholders, and to provide information to help stakeholders to help advance drug development. Common CMC Issues for Phase 1 IND Study. Other Questions.

Reagent 59

Reagent Manufacturing Production Gene Therapy 59

Delveinsight

AUGUST 20, 2020

FDA rejects BioMarin’s gene therapy and Gilead’s filgotinib over concerns. The FDA refused to approve BioMarin’s hemophilia A gene therapy valoctocogene roxaparvovec. The removal of an AdComm was seen as a positive. J&J has agreed to pay USD 52.50

Gene Therapy 52

Gene Therapy Research Gene Antibody 52

pharmaphorum

APRIL 15, 2021

Roche’s Genentech unit has released further data from a key trial of Evrysdi, its oral treatment for spinal muscular atrophy (SMA), the rare muscle wasting disease that is often fatal in babies that develop it. Roche leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics.

Gene Therapy 52

Gene Therapy Protein Clinical Development Regulation 52

Camargo

AUGUST 11, 2021

As regulatory requirements become increasingly harmonized across the globe, the development and marketing of pharmaceutical products worldwide are also becoming more streamlined. As of January 1, 2021 , EU pharmaceutical regulations do not apply to the United Kingdom, which formally left the EU in January 2020.

Production 230

Production Packaging Packaging Marketing 230

The Pharma Data

JANUARY 7, 2021

As CEO of Freeline, a liver-directed gene therapy company, she scaled the company from preclinical stage to a fully integrated biotechnology organization, which included a broad, internally developed pipeline, two programs in clinical development and a commercial-scale, high-quality CMC and manufacturing platform.

Gene Therapy 52

Gene Therapy Life Science Production Clinical Development 52

Drug Discovery World

SEPTEMBER 14, 2023

joined Pfizer’s Executive Leadership Team as Chief Oncology Research and Development Officer and Executive Vice President reporting to Chairman and Chief Executive Officer, Albert Bourla. In July 2023, AstraZeneca and Ionis Pharmaceuticals expanded their existing collaboration on eplontersen to also include Latin America.

Drugs 59

Drugs Vaccination Vaccine Medicine 59

Advarra

FEBRUARY 24, 2023

How do you strategically plan your therapy and research objectives to improve the chances you will satisfy what the regulators are looking for and ultimately get your therapy approved for marketing? Sponsors need to carefully consider how they will prove safety and efficacy in a manner sufficient to satisfy the regulators.

Clinical Trials 52

Clinical Trials Clinical Trials Trials Regulation 52

pharmaphorum

NOVEMBER 27, 2020

“Key factors have been the emergence of communities around some of these diseases that have driven major legislative change and ensured that regulators are more flexible.”. Bringing the patients and their families into the conversation early does help to define the clinical development path.”. Shifting tide. Market research.

Development 132

Development Clinical Trials Clinical Trials Drugs 132

The Pharma Data

DECEMBER 17, 2020

Cadavid will be responsible for the strategy, direction and execution of the company’s clinical development programs. Prior to Fulcrum, Cadavid held several leadership positions at Biogen, including Senior Medical Director of the multiple sclerosis clinical development group. Immune Regulation – U.K.-based

Development 52

Development Gene Therapy Engineer Life Science 52

XTalks

DECEMBER 24, 2020

From rare disease drug approvals to treatments involving immunotherapies and gene therapies and awarding of a Nobel Prize to the inventors of the gene-editing tool CRISPR, 2020 was a year of great activity and productivity despite the backdrop of the pandemic. Vaccine Considerations.

Life Science 111

Life Science Vaccination Vaccine Gene 111

Drug Discovery World

JUNE 16, 2023

The International Society for Cellular Therapy (ISCT) is a global society of clinicians, regulators, technologists, and industry partners with a shared mission to drive the translation of all cell and gene (CGT) therapies for the benefit of patients worldwide.

Manufacturing 52

Manufacturing In-Vivo Production Compliance 52

XTalks

OCTOBER 18, 2022

A clinical research organization (CRO) is a company that delivers outsourced services to plan, manage and execute clinical trials for biotechnology, pharmaceutical, and medical device companies. Cellular and gene therapy. Radiation therapy. European Union (EU) regulations. Hematology and oncology.

Clinical Research 111

Clinical Research Research Clinical Trials Clinical Trials 111

pharmaphorum

APRIL 28, 2022

The new hub will focus on centralising things like clinical development strategy, clinical operations, and regulatory approval strategy – not necessarily drug development itself. How big pharma tackles rare diseases. But they also don’t have to invest in mass market advertising.

Pharma Companies 59

Pharma Companies Branding Development Marketing 59

pharmaphorum

APRIL 15, 2021

We have a pipeline of novel therapeutics and technological approaches, advancing a diverse array of candidates, including small molecules, biologics and cell and gene therapies, so a lot of the time we are working where there’s little, or no precedent.”. It’s through this collaboration that we help shape the future.”.

Development 62

Development Manufacturing Production Bioinformatics 62

Advarra

AUGUST 10, 2023

Rapid growth in gene therapy is expected to receive additional support as the Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER) prepares to launch Operation Warp Speed for Rare Diseases. Peter Marks, head of FDA’s CBER – the organization responsible for regulating gene therapies.

Gene Therapy 52

Gene Therapy Development Gene Drugs 52

FDA Law Blog

SEPTEMBER 20, 2021

We have seen first-hand the difficult task faced by CBER review staff to keep up with the flood of innovative cell and gene therapy products, as well as the taxing influx of COVID-19 pandemic related applications requiring review resources in both Centers. Notable Dates and Timelines – Applications open, Q4 of FY 2023.

Development 52

Development Gene Therapy Production Drugs 52

Drug Discovery World

JULY 13, 2023

The rules and regulations Unlicensed drugs are drugs that have not yet been assessed by a regulator, while off-label use is when an approved medication is prescribed outside of the terms of its license, e.g., for a different disease or in a different patient group.

Drugs 94

Drugs Medicine Regulation Licensing 94

The Pharma Data

DECEMBER 9, 2020

The Chinese biopharmaceutical industry is growing in leaps and bounds, but there is still a huge unmet need when it comes to getting patients access to the breakthrough therapeutic modalities and platforms like RNAi, cell and gene therapy and others. A solution could be at hand with Overland Pharmaceuticals.

In-Vivo 52

In-Vivo In-Vitro Antibody Development 52