CDMO eXmoor Pharma and biotech company Evox Therapeutics have announced the completion of a technology transfer programme for Evox's exosome-enabled gene editing technology and specifically its MSH3-targeted programme for the treatment of Huntington’s disease.
Evox's pipeline includes next-generation gene editing medicines that target genetically driven neurodegenerative diseases, including Huntington's disease and amyotrophic lateral sclerosis.
The programme involved the transfer and establishment of Evox’s proprietary manufacturing process (which pairs the natural transport abilities of exosomes with genetic editors and therapeutic payloads) and associated analytical methods at eXmoor’s Bristol facility.
The pair said that the completion of this phase provides the "technical foundation for subsequent transfer into GMP and future clinical manufacture."
Exosome-based gene editing therapeutics utilise small, naturally occurring cellular vesicles (exosomes) to deliver gene-editing tools such as CRISPR/Cas9 into targeted cells.
This approach safely moves through the body, avoiding immune system defences to correct faulty genes and address diseases at their source.
Researchers and biotech companies are actively advancing these therapeutics to address difficult-to-reach genetic disorders.
The duo added that the programme reflects the growing need to translate novel platforms into scalable manufacturing processes capable of supporting clinical development.
Lucy Foley, CEO of eXmoor Pharma, said: "Translating a novel platform of this kind into a robust and manufacturable process requires process development, analytical development and manufacturing expertise to be fully integrated from the outset."
The successful establishment of Evox’s process and analytical methods at our Bristol facility reflects the strength of the collaboration between our teams and provides a clear foundation for the next stage of the programme.
Per Lundin, CEO of Evox Therapeutics, added: "As we continue to advance our pipeline of next-generation gene editing medicines, establishing strong technical and manufacturing foundations is an important part of our development strategy."
eXmoor Pharma’s multidisciplinary expertise and collaborative approach made them the right partner to support this work as we progress our platform and Huntington’s disease programme towards clinical development.
