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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. There are also nuances for operational planning in cell therapy development programs.
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. Under the deal, Rentschler Biopharma’s ATMP facility in Stevenage, UK, will undertake the bioprocess development of adeno-associated virus (AAV) material.
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.
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. at the BioProcess International Theater.
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UK-based Oxford Biomedica has signed a new development, manufacture and license agreement with Beam Therapeutics for next-generation CAR-T therapeutics. The agreement also puts in place a three year clinicalsupply agreement for the two companies.
Food and Drug Administration (FDA) has granted Fast Track designation to VTX-801, Vivet’s clinical-stage gene therapy for the treatment of Wilson Disease – a rare, genetic disorder that reduces the ability of the liver and other tissues to regulate copper levels, causing severe hepatic damage, neurological symptoms, and potentially death.
billion on offer in milestones if the programmes meet development and commercial objectives. According to a statement from the two companies, Arcturus will contribute its STARR self-amplifying mRNA vaccine and LUNAR lipid nanoparticle delivery platform, which has already been deployed in the development of COVID-19 vaccine ARCT-154.
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.
This could be seen as a positive for companies developing CAR T-cell therapies for autoimmune diseases and solid tumors. According to Carvykti co-developer Legend Biotech, across US treatment centers that offer both therapies, Carvykti holds around 80 percent of the late-line market share.
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