Drug Discovery World

FEBRUARY 1, 2024

Vivet is a clinical stage biotech company developing gene therapies for rare inherited metabolic disorders. CrisTauX will enable accelerated clinical development of VTX-806 upon completion of the programme. Vivet Therapeutics has received €4.9

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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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Pharmaceutical Technology

NOVEMBER 21, 2022

RVAC Medicines has signed a master research partnership agreement with the Agency for Science, Technology and Research (A*STAR) for analysing and developing solutions to build messenger ribonucleic acid (mRNA) manufacturing and analytics expertise in Singapore. Topic sponsors are not involved in the creation of editorial content.

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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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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 therapy was also recently approved by the FDA for patients in the US. .

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Drug Discovery World

SEPTEMBER 6, 2022

By Dr. Deborah Phippard, Chief Scientific Officer of Precision for Medicine and Anshul Mangal, President & General Counsel of Project Farma. . In recent years the advanced therapy industry has been the most significant disruptor in medicine. ViaCyte, Beam, and Verve are all due to begin gene editing trials this year.

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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.

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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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The Pharma Data

JUNE 7, 2023

Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics Bayer AG is joining forces with Acuitas Therapeutics, Inc., a biotechnology company specializing in the development of lipid nanoparticle (LNP) delivery systems for molecular therapeutics.

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FDA Law Blog

NOVEMBER 2, 2023

Lewis, Senior Regulatory Device & Biologics Expert — On October 20, 2023, FDA announced the availability of the final guidance authored by CBER titled “Voluntary Consensus Standards Recognition Program for Regenerative Medicine Therapies.” It finalized a draft guidance published in 2022.

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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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Pharmaceutical Technology

AUGUST 24, 2022

Additionally, the AAV capsid platform of Lacerta will be leveraged for discovering and developing new AAV capsids that are streamlined for acting on preferred CNS tissues and cell types. Prevail will also handle the complete production, clinical development and marketing activities.

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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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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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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.

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XTalks

OCTOBER 4, 2023

Heather McDonald: My previous role at Bayer was a global-level role with a specific focus on cell and gene therapies. In addition, AstraZeneca just announced a major acquisition of Pfizer’s cell and gene therapy portfolio. How is the shift toward value-based healthcare affecting drug pricing and reimbursement?

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Pharmaceutical Technology

FEBRUARY 8, 2023

Under the deal, AGC Biologics will be responsible for manufacturing the cell therapy lentivirus-based product for the ongoing clinical programmes of Genenta Science. Genenta already had an existing MSA with Molecular Medicine’s facility in Milan, which was acquired in 2020 by AGC Biologics.

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Pharma Marketing Network

DECEMBER 21, 2020

Almost two decades after the human genome was sequenced, a trickle of new genetic medicines (i.e., those that modify the expression of an individual’s genes or repair abnormal genes) has entered clinical practice, including 11 RNA therapeutics, 2 in vivo gene therapies, and 2 gene-modified cell therapies.

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Pharmaceutical Technology

AUGUST 17, 2022

The latest development comes after the company and Cerevance signed a multi-year strategic research collaboration to discover new targets for Alzheimer’s disease. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. Topic sponsors are not involved in the creation of editorial content.

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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.

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Drug Discovery World

FEBRUARY 10, 2023

The research will support the clinical development of CTx001, CTx’s lead gene therapy asset for the treatment of GA, by enabling the identification of patients with complement-driven AMD for future interventional studies.

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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. Adjusting for COVID-19.

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Pharmaceutical Technology

AUGUST 4, 2022

Poseida will carry out the Phase I clinical trials and produce clinical materials for a subset of both the company’s portfolio programmes licensed or optioned to Roche and the companies’ future partnership programmes, before transferring the programmes to the latter for additional development and marketing.

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pharmaphorum

OCTOBER 25, 2022

If completed, the acquisition – which has been termed a “low ball bid” by some – of AGTC will become Syncona’s third company focused on retinal gene therapy (after Nightstar and Gyroscope , acquired by Biogen and Novartis, respectively). which develops innovative therapeutics for neurotology. million in cash ($0.34 per share).

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The Pharma Data

JANUARY 17, 2021

18, 2021 /PRNewswire/ — Aruvant Sciences, a private company focused on developing gene therapies for rare diseases, and Lonza announced today their agreement in support of ARU-1801, Aruvant’s one-time investigational gene therapy for sickle cell disease (SCD). NEW YORK , Jan.

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Drug Discovery World

OCTOBER 4, 2022

This year’s event will cover some of the most exciting advancements in areas spanning screening, automation, high content imaging, disease models, cell and gene therapies and how innovation is being driven through partnerships and collaborations. Cell and gene therapies. billion, compared to $19.9

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XTalks

JANUARY 17, 2024

There are many aspects of oncology drug development that are driven by the unique nature of the treatments being developed and the needs of the patients, their caregivers and the doctors that treat them.

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Camargo

JULY 30, 2021

In 1981, I got my doctorate degree in Clinical Pharmacology, but more importantly, my research was heavily involved in neonatal medicine, developing drugs there. What would you say have been the most significant changes to pediatric drug development and clinical research in that time?

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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.

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The Pharma Data

DECEMBER 31, 2020

where he led global clinical development programs including clinical strategy, clinical development, and regulatory affairs. Prior to Taiho, he held the positions of senior vice president of Clinical Oncology and CMO at Geron Corporation. and CMO for Taiho Oncology, Inc.

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The Pharma Data

JANUARY 26, 2021

Short will be responsible for overseeing the clinical development of VarmX’s pipeline, including progression of its lead product, VMX-C001, into clinical proof of concept and registrational studies. During this time, he grew the medical, clinical, and regulatory groups, and established an in-house pharmacovigilance function.

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Drug Discovery World

SEPTEMBER 14, 2023

Excluding contributions from Comirnaty and Paxlovid, company revenues grew $537 million, or 5%, operationally. AstraZeneca saw total revenue reach $22,295m, up 4% despite a decline of $2,181m from Covid-19 medicines. Excluding Covid-19 medicines, total revenue increased 16% and product sales increased 15%. Specialty care grew 11.8%

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Velocity Clinical Research

OCTOBER 4, 2023

Shilpa Muthusamy, MD is an experienced clinical research physician, and holds a Bachelor of Medicine and Bachelor of Surgery from the University of Nottingham. Matt Fisher, MD qualified from Bristol Medical School in 2015 and worked as a doctor in general and emergency medicine for five years in the NHS.

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The Pharma Data

APRIL 27, 2022

We plan to discuss these data with health authorities, and we will continue to expand our tislelizumab clinical development program in pursuit of novel, synergistic combinations with the ultimate goal of extending survival for more patients.”. About Novartis Novartis is reimagining medicine to improve and extend people’s lives.

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pharmaphorum

OCTOBER 13, 2022

million in future payments tied to preclinical, clinical development, and commercial achievements. At the end of last year, the company partnered with Atara on mesothelin-targeted CAR-T cell therapies for solid tumours. Bayer bought Vividion for $1.5 Vividion is making an upfront payment of $17.5

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XTalks

MAY 9, 2024

As we explore Genmab’s strategic vision, technological advancements and the next frontier in antibody research, we gain insights into the future of medicine — a future where Genmab’s cutting-edge therapies are poised to transform patient care and meet the complex challenges of the next generation of healthcare.

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The Pharma Data

DECEMBER 10, 2020

Dr. Pirozzi received a Doctorate of Medicine from Università Campus Bio-Medico di Roma in Italy, a Doctorate of Philosophy in Immunology from Sapienza Università di Roma in Italy, a Post-Doctorate degree in Immunology from the Institut Pasteur in Paris, France, and business training at the London Business School, UK.

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