“Gene therapy investments and partnerships signal substantial shift in partnering approach and strategy. In stark departure from the common preference for late stage clinical assets, several leading pharma companies have demonstrated appetite for early stage investments and upstream enabling technologies across R&D and manufacturing scale-up. These pave the way for stronger research ecosystems being anchored by these investors and potentially emerging as more competitive partners for the highly contented preclinical and clinical stage gene therapy assets. The upstream partnerships trend has several deals to boot – Oxford BioMedica and PhoreMost, Roche and Cevec, Eli Lilly and Precision Biosciences, UCB and Handl, and the big Bayer – Asklepios $4 billion deal.”

Corporate momentum in newer-age therapies had historically been entrenched in mid to late stage assets with substantial clinical and scientific validation with respect to clinical use-case and benefit of the therapy. However, strategies to emerge as an entrenched contender in the evolving cell and gene therapy space has resulted in rescripting of biopharma deal making appetite. This expanding partnership interest comes at a time when pioneering gene therapy drugs have been approved by regulators and are gaining commercial traction. Investment allocation is at an all-time high. The cell and gene therapy segment is witnessing the trend of big pharma breaking out of the mould of late stage clinical asset licensing/acquisition. Partnerships and acquisitions have spanned the breadth of early stage assets, technology platforms and upstream facilitative technologies.

A large portion of the science in the cell and gene therapy has emerged out of academia or smaller biotechnology companies. While the larger trend of research externalization by big pharma continues, it is resulting in a broader ecosystem in the case of this cell and gene therapy. Current slew of pharma contenders in cell and gene therapy are now rapidly making deals to establish capability in both upstream technology platforms research and downstream manufacturing capacity. Overall, they will then able to accelerate assets that they license into the portfolio and even emerge as more competitive partners for these highly contented preclinical and clinical stage assets. We are excited about the implications of this trend for more rapid development of the overall research ecosystem for next generation therapies.

The month of November saw some notable deal-making in the space, beginning with Oxford BioMedica and PhoreMost joining hands to develop next-gen cell therapies. PhoreMost will deploy its proprietary SITESEEKER® platform to identify potential therapeutic candidates for Oxford Biomedica’s LentiVector® gene therapy delivery system. Oxford’s LentiVector® system delivered the world’s first approved CAR-T cell therapy Kymriah and now Oxford seeks to deploy the platform to develop newer therapies for unmet diseases. More details on the deal can be found in our PharmForward post here.

Roche has actively been partnering for both upstream research platforms and gene therapy manufacturing platform to establish an integrated R&D and manufacturing engine. After having partnered with Dyno Therapeutics last month for developing synthetic AAV capsids directed towards central nervous system and liver disorders, Roche has now tapped into Cevec’s ELEVECTA® gene therapy manufacturing technology. Cevec’s platform utilizes helper-virus free stable producer cell lines with all components for AAV production integrated into the cell line, which ensures low batch-to-batch variability, superior vector quality and convenient scalability. The patent protected technology overcomes some of the challenges associated with current manufacturing technologies with its superior scalability, product quality and process stability. More on the transaction can be found here in our PharmForward post.

UCB acquired Handl Therapeutics to develop in vivo gene therapy to treat complex neurodegenerative diseases. Simultaneously, UCB has also partnered with Lacerta Therapeutics to utilize its proprietary AAV capsid technology platform and scalable manufacturing platform for developing AAV-based therapies. The acquisition comes closely on the heels of Bayer’s USD 4 billion acquisition of Asklepios Biopharmaceutical Inc. specializing in developing gene therapies across different therapeutic areas including neuromuscular, Central Nervous System (CNS), cardiovascular (CVS) and metabolic diseases.

The last week of the month also saw another deal from a big pharma, Eli Lilly, for collaborating to develop various gene therapies. Eli Lilly announced research collaboration and exclusive license agreement with Precision BioSciences to utilize its proprietary ARCUS® platform for the research and development of in vivo therapies for three genetic disorders, primarily focused on Duchenne Muscular Dystrophy. The ARCUS® platform is based on a naturally existing gene editing homing endonuclease I-CreI which allows to make highly specific edits and has a substantial benefit over other gene editing platforms in the sense that it comes with a built-in safety switch to prevent any unwanted off-target DNA edits – a major limitation and clinical risk inherent to many gene editing platforms. The deal comes closely after Ultragenyx partnered with Solid Biosciences, in late October, for the development of gene therapies for Duchenne Muscular Dystrophy, combining micro dystrophin construct developed by Solid Biosciences and Ultragenyx’s HeLa producer cell line (PCL) manufacturing platform and Adeno Associate Virus 8 (AAV8) variants.