pharmaphorum

MAY 23, 2024

Merck KGaA has agreed to a $600m deal to buy Mirus Bio, a specialist in transfection reagents used in the production of cell and gene therapies.

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Pharmaceutical Technology

MAY 24, 2023

Forge Biologics has joined the public-private collaboration, the Bespoke Gene Therapy Consortium (BGTC), to expedite the development and manufacture of new AAV [adeno-associated virus] gene therapies to treat patients with rare diseases. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Pharmaceutical Technology

MARCH 10, 2023

On 10 March, the National Health Service Blood and Transplant (NHSBT) opened a new Clinical Biotechnology Centre (CBC) with the aim of improving the UK’s ability to develop and manufacture cell and gene therapies. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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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.

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Bio Pharma Dive

MAY 21, 2021

The treatment, for a progressive, often deadly brain disease, could soon become Bluebird's third approved product and one of only a handful of marketed gene therapies in the world.

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Pharmaceutical Technology

MAY 25, 2023

ElevateBio has raised $401m in a Series D financing round for advancing its technology platforms to expedite the design, production and development of cell and gene therapies. ElevateBio intends to use the funds to advance its genetic medicine current good manufacturing practice (cGMP) and process development business, BaseCamp.

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Bio Pharma Dive

APRIL 11, 2023

Founded by University of Pennsylvania researchers, VintaBio will manufacture the viral vectors that are essential in cell and gene therapy production, but which have been in shorter supply as more companies entered the space.

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Pharmaceutical Technology

MAY 16, 2023

The Foundation for the National Institutes of Health (FNIH) has announced its plans to prioritise eight rare diseases to provide industry standards for manufacturing, preclinical testing and product analytical testing for gene therapy development.

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Pharmaceutical Technology

JANUARY 5, 2023

Capsida Biotherapeutics and Eli Lilly and Company ’s wholly owned subsidiary Prevail Therapeutics have announced a partnership for the development of non-invasive gene therapies for central nervous system (CNS) diseases. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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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.

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Pharmaceutical Technology

MAY 24, 2023

It can correct several disease elements associated with PKD, a rare, monogenic red blood cell disorder caused by a mutation in the PKLR gene. It can correct several disease elements associated with PKD, a rare, monogenic red blood cell disorder caused by a mutation in the PKLR gene.

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Pharmaceutical Technology

JUNE 19, 2023

Although only a small number of gene therapies have reached the market thus far, the industry is poised to grow quickly over the next few years. According to GlobalData’s clinical trials database, there are currently 1,231 planned and ongoing trials for gene therapies and gene-modified cell therapies alone.

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Pharmaceutical Technology

APRIL 25, 2023

Forge Biologics has received a qualified person (QP) declaration to manufacture adeno-associated virus (AAV) gene therapies to support European clinical programmes. The FBX-101 is an AAV serotype rh10 gene therapy, given intravenously following haematopoietic stem cell transplantation infusion.

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Fierce Pharma

MARCH 14, 2024

After charting multiple personalized medicine expansions in recent years, contract manufacturer Charles River Laboratories is back with another gene therapy pact—this time centered on an adeno-asso | After charting multiple personalized medicine expansions in recent years, contract manufacturer Charles River Laboratories is back with another gene therapy (..)

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Pharmaceutical Technology

DECEMBER 14, 2022

Merck and Synplogen have signed a non-binding Memorandum of Understanding (MoU) to expedite the development and manufacturing of viral vector-based gene therapy applications. The firms intend to merge their expertise to provide simplified viral vector gene therapy development, production and testing in Japan.

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Pharmaceutical Technology

JUNE 2, 2023

Rentschler Biopharma has collaborated with Ikarovec for expediting the new gene therapies to treat ophthalmic disease. The company is developing new and differentiated gene therapies for the treatment of serious, but common eye diseases. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Pharmaceutical Technology

NOVEMBER 6, 2023

GlobalData uncovers the leading innovators in rAAV production technology for the pharmaceutical industry.

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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. Enhancing Product Quality Reviews.

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XTalks

JUNE 8, 2023

Over the past few years, there has been a significant expansion in the cell and gene therapy landscape, with an increasing number of therapies entering clinical trials and receiving regulatory approvals. “I think if we all go into these relationships with this mindset, we’ll achieve great things together.”

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Pharmaceutical Technology

JUNE 8, 2023

GenScript ProBio has collaborated with the New York Blood Center Enterprises’ business unit, called Comprehensive Cell Solutions (CCS), to accelerate the development and manufacture of cell and gene therapies. The core objective of the collaboration is to make the therapies affordable and accessible to patients.

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Pharmaceutical Technology

APRIL 13, 2023

Two gene therapies up for approval this year for sickle cell disease could be cost effective in some cases at a $2 million price point, based on a draft evidence report published by the Institute for Clinical and Economic Review (ICER). Also known as lovo-cel, bluebird bio’s product is a lentiviral gene therapy.

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Gene Therapy Gene FDA Approval Licensing 147

Fierce Pharma

FEBRUARY 26, 2024

Not even one year after becoming a commercial biotech, Krystal Biotech has blockbuster dreams for its flagship product. | In its first six months of commercial availability, Krystal's topical gene therapy Vyjuvek raked in more than $50 million.

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Pharmaceutical Commerce

MAY 6, 2024

In an interview with Pharma Commerce Editor Nicholas Saraceno, Martin Hawes, Global Head of Product Development, Tower Cold Chain offers insights on the future of cell & gene therapies when it comes to cold chain storage.

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Pharmaceutical Technology

OCTOBER 14, 2022

Pharmaceutical companies Xcell Biosciences (Xcellbio) and aCGT Vector have partnered to expedite the development of cell and gene therapies. The two companies will aim to improve the manufacturing and analytic procedures used to develop personalised cell and gene therapies to treat cancer patients.

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Drug Discovery World

JUNE 23, 2023

Sarepta Therapeutics’ Elevidys has become the first gene therapy for Duchenne muscular dystrophy (DMD) to gain marketing authorisation in the US. It is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene.

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BioSpace

MAY 22, 2024

BioSpace's Lori Ellis discusses the risks and challenges of cell and gene therapy combination products with DIA speakers James Wabby, AbbVie and Rob Schulz, Suttons Creek, Inc.

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Drug Discovery World

NOVEMBER 16, 2023

The Medicines and Healthcare products Regulatory Agency (MHRA) has authorised the world’s first gene therapy for sickle-cell disease (SCD) and transfusion-dependent β-thalassemia (TDT). Casgevy is designed to work by editing the faulty gene in a patient’s bone marrow stem cells so that the body produces functioning haemoglobin.

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XTalks

DECEMBER 13, 2023

The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. Both therapies will be available in early 2024.

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BioPharma Reporter

JUNE 30, 2021

Cobra Biologics, the gene therapy division of the Charles River owned Cognate BioServices, and Sweden's CombiGene recently announced the successful production and quality assurance of the plasmids to be used in the GMP production of the gene therapy, CG01.

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XTalks

MAY 17, 2023

The Foundation for the National Institutes of Health (FNIH) announced this week that the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC) has selected eight rare diseases for its clinical trial portfolio. As such, rare disease patients and their families often face little hope for effective treatments.

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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. Around 40% of clinical holds are for gene therapy programs.

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Bio Pharma Dive

SEPTEMBER 17, 2022

The therapy, called Skysona and cleared to treat cerebral adrenoleukodystrophy, is the product of more than a decade of work by Bluebird. It will cost $3 million.

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Drug Discovery World

DECEMBER 8, 2023

Andrew Busey , Co-Founder, Form Bio discusses why vertical AI is set to reshape the cell and gene therapy sector. The cell and gene therapy industry is no exception to this digital transformation. Here’s what this means for pharmaceutical leaders and patients.

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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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Pharma Mirror

APRIL 28, 2021

The post AavantiBio and Catalent Announce Partnership to Support Development and Manufacturing of Gene Therapies for Rare Genetic Diseases appeared first on Pharma Mirror Magazine. and Europe. Catalent will further support process optimization and look to reduce material.

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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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Drug Discovery World

FEBRUARY 8, 2024

In 2023, DDW hosted a webinar, ‘A comprehensive guide to using adeno-associated virus (AAV) vectors in gene therapy’, supported by Azenta Life Sciences. AAV is optimal for gene therapy due to its efficiency and safety in humans; however, AAV processes can present obstacles for researchers. How is the titer determined?

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