Cell and Gene Therapy Catapult and pHion Therapeutics announce licencing agreement for RALA technology

Collaboration could reduce the cost of cell and gene therapy production by developing a more efficient method of modifying cells for therapies

The Cell and Gene Therapy (CGT) Catapult and pHion Therapeutics today announce an exclusive licence agreement for pHion’s proprietary RALA technology for ex-vivo applications.

The agreement builds upon the September 2017 collaboration between the organisations and aims to build alternative platforms for gene delivery and lower the costs of goods (COG) for cell therapy manufacturing.

The CGT Catapult will develop good manufacturing practice (GMP)-compliant processes to use RALA, a charged peptide that spontaneously forms nanoparticles when mixed with nucleic acids, as a way to develop and supply ex vivo cell and gene therapies where the end product is a modified cell or virus.

These processes will then be used by pHion to help promote the research use of RALA in the development of new therapies.

One of the major costs in cell and gene therapy is the ex-vivo delivery of genetic material into the cells by means of a virus, this can account for up to half of the COG. Utilising RALA, a non-viral delivery methodology instead, could significantly reduce the COG and dependence upon viral vectors.

The design of cost effective, non-viral vehicles, capable of transporting and releasing functional genetic material at high efficiencies without toxicity is one of the main challenges for the development of gene-modified cell and gene therapies.

Under the agreement, CGT Catapult will be able to sub-licence the use of the technology with pHion developing the upstream RALA nucleic acid package for the GMP process. pHion and CGT Catapult will then promote the technology for the research and development of processes and protocols to develop new cell and gene therapeutics.

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Commenting on the collaboration, Keith Thompson, Chief Executive Officer of CGT Catapult, said: “The potential of RALA technology for decreasing the costs of production of cell and gene therapies is significant. We are proud to support and accelerate the growth of this exciting new UK company by providing pHion access to our knowledge and expertise in cell and gene therapies."

“Our expectation is that a RALA-based manufacturing process, alongside appropriate integration technologies where required, will give companies an advantage in the highly competitive immunotherapy market.”

Professor Helen McCarthy, Founder and Chief Executive Officer of pHion Therapeutics added: “This first of its kind licence agreement for pHion and CGT Catapult allows us to build on our existing successful collaboration."

"We firmly believe pHion’s RALA technology will be disruptive for ex vivo cell and gene therapies and lower COG for Cell Therapy manufacturing. Our partnership with CGT Catapult will allow us push the technology forward in this area and deliver on this."

"We are excited to continue to work with CGT Catapult to develop pHion’s manufacturing processes to GMP standard and to work with CGT Catapult’s network of companies to develop new CAR-T or T-cell receptor-based therapeutics utilising the RALA technology.”

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