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By Amy Raymond, PhD, PMP, Executive Director, Therapeutic Strategy Lead, Rare Disease Cell and gene therapies (CGTs) include cutting-edge approaches that offer the hope of a healthier, happier, and better tomorrow for a wide range of patient populations. The global nature of cell and gene therapy trials adds another layer of complexity.
By Luisa Sterkel & Joana Loureiro , Tenthpin Consultants The promise and potential of cell and gene therapies (CGT) has emerged in the recent past and currently over 1.500 CGT are registered for clinical trials holding great hope for the treatment of challenging and uncurable diseases.
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.
Contract packaging and clinicalsupply services company Sharp has completed the construction of new purpose-built production suites to facilitate the packaging, storage and distribution of gene therapies at its facility in Heerenveen, The Netherlands.
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.
The expansion forms part of Catalent’s ongoing global strategy to increase its ability to handle, store and manage advanced therapies for clinicalsupply, and follows investments at its facilities in Philadelphia, Singapore, and Shanghai, China, in specialized, ultra-low temperature storage capabilities. With sites in the U.S.,
Contract development and manufacturing organization, Catalent, has invested in the capabilities at its clinicalsupply services facility in Philadelphia, in the US, to further support biotech firms developing cell and gene therapies.
The agreement also puts in place a three year clinicalsupply agreement for the two companies. Under the terms of the deal, Oxford Biomedica will receive an undisclosed upfront payment, with additional payment related to development and manufacturing of lentiviral vectors for use in clinical trials. .
Vivet Therapeutics (“Vivet”), a clinical-stage biotechnology company, and Pfizer Inc. VTX-801 is a novel investigational gene therapy to be evaluated in a Phase 1/2 clinical trial to determine the safety, tolerability, and pharmacological activity of a single intravenous infusion in adult patients with Wilson Disease.
“This collaboration is an exciting opportunity to complement CSL’s own next-generation mRNA programme with a partner who developed a platform to deliver late stage clinicalsupplies at scale,” said CSL’s chief operating officer Paul McKenzie.
Originally launched in 2019 for early-phase protein therapy development, OneBio Suite offers customers an integrated service to accelerate programs from development to manufacturing, including fill/finish and packaging, and support for clinicalsupply and commercial launch.
BioNTech plans to manufacture additional therapeutic and vaccine drug candidates at the plant, such as other mRNA vaccines, antibody and cell and gene therapy candidates to support the development of its diversified cancer and infectious disease product pipeline.
The companies have also expanded a contract manufacturing agreement with Novartis that will help produce Carvykti for both commercial and clinicalsupply at the New Jersey plant through the end of 2029. 2seventy has been used to the struggles ever since it spun out of gene therapy biotech bluebird bio.
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