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.

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Drug Discovery World

JANUARY 5, 2023

CANbridge Pharmaceuticals has exercised its option to secure the exclusive global rights to develop, manufacture and commercialise a novel gene therapy to treat spinal muscular atrophy (SMA) from UMass Chan Medical School. .

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XTalks

JANUARY 3, 2024

Novartis and Voyager Therapeutics have struck a licensing deal that will see Novartis pay Voyager $100 million upfront to develop gene therapy candidates targeting Huntington’s disease (HD) and spinal muscular atrophy (SMA). billion with preclinical, development, regulatory and sales milestones. Sandrock, Jr.,

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Roots Analysis

FEBRUARY 27, 2024

Over the last few decades, various viral vector manufacturing have been developed, optimized and standardized for introduction of therapeutic DNA / gene of interest into a patient’s body / cells. On the other hand, among non-viral gene delivery tools, plasmid DNA has emerged as the preferred option.

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Pharmaceutical Technology

FEBRUARY 8, 2023

Genenta Science and AGC Biologics have signed a development and manufacturing service agreement (MSA). Under the deal, AGC Biologics will be responsible for manufacturing the cell therapy lentivirus-based product for the ongoing clinical programmes of Genenta Science.

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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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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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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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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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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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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

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.

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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? Curbishley explains that as the wave of new therapies in development continues to increase, global pressures on manufacturing availability will become more acute.

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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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Drug Discovery World

SEPTEMBER 20, 2022

As a condition of the SKYSONA Accelerated Approval, the manufacturer will provide confirmatory long-term clinical data to the FDA. 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

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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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pharmaphorum

JANUARY 11, 2021

While bluebird has conducted pioneering work in gene therapy for blood disorders and in cancer cell therapy, products have been delayed by issues with filing data for the FDA. The post bluebird bio to split into oncology and gene therapy specialists appeared first on. million price tag. million price tag.

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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. In a paper published in Molecular Therapy: Methods & Clinical Development , the team discussed why they choose this type of gene therapy to treat HIV.

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Drug Discovery World

SEPTEMBER 28, 2023

Can a manufacturer of a cell or gene therapy switch suppliers midstream in clinical development? What are the costs and time delays of switching suppliers later in clinical development? How will not having a GMP supplier impact the speed a therapy gets to market?

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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

APRIL 5, 2023

Caribou Biosciences chief medical officer Syed Rizvi said: “Fast Track designation for CB-011 allows us instrumental interactions with the FDA as we progress our clinical development and regulatory plans for CB-011. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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The Pharma Data

JANUARY 12, 2021

Marianne De Backer, Head of Business Development & Licensing in Bayer’s Pharmaceuticals Division, pictured above. Also in December, the company partnered with Atara Biotherapeutics to develop off-the-shelf T-cell immunotherapies for high mesothelin-expressing tumors. Photo courtesy of Bayer. We have set the bar high.

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Drug Discovery World

JUNE 16, 2023

Finally, this article covers the CGT trends when it comes to redefining manufacturing. Manufacturing processes are among the bottlenecks of CGT wider use. Clarke thinks that data and digitisation are the keys to unlocking manufacturing. Next, attendees discuss the trend of allo vs auto.

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XTalks

OCTOBER 22, 2020

To achieve transparency and actionability of data, pharmaceutical companies are evolving their manufacturing processes by digitalizing the data from all of their equipment. At Aizon , we provide solutions to speed up pharmaceutical manufacturing process development from the start in a GxP compliant platform.

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The Pharma Data

SEPTEMBER 22, 2020

Novartis Gene Therapies to initiate new pivotal confirmatory study to evaluate use of AVXS-101 intrathecal (I T ) formulation in older patients with SMA to further support registration. Novartis Gene Therapies remains confident in the overall benefit-risk profile for patients on treatment.

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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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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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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.

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The Pharma Data

JANUARY 27, 2021

Food and Drug Administration (FDA) has cleared an investigational new drug (IND) application for PBFT02, an adeno-associated virus (AAV)-delivery gene therapy that is being studied for the treatment of patients with Frontotemporal Dementia (FTD) with granulin (GRN) mutations. chief executive officer of Passage Bio. “FTD

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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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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

This approach remains logistically complex, which builds the desire for allogeneic treatments that can be manufactured from unrelated donors on a larger scale. But while companies continue studying allogeneic CAR-T therapies, including for their coveted use in solid tumours, such advancements remain challenging.

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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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Delveinsight

JULY 28, 2020

Moderna has initiated the first large-scale Phase III clinical trial of its COVID-19 vaccine , mRNA-1273 after an expanded contract with the Biomedical Advanced Research and Development Authority (BARDA). At present, more than 150 vaccine candidates are in various stage of clinical development against SARS-CoV-2.

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Roots Analysis

FEBRUARY 3, 2023

It is also worth highlighting that there are a significant number of cell therapies that are currently under clinical development. Several industry players (XX) are actively contributing to the manufacturing and development of cell therapies in North America, Europe and Asia Pacific regions.

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Drug Discovery World

FEBRUARY 12, 2024

Leslie Orne, President and CEO, Trinity Life Sciences “AI and ML will play an increasingly important role in the clinical development and commercialisation of cell and gene therapies, which are typically targeted to small patient populations. Looking even further into the future, Industry 4.0

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