AmorChem invests in a new gene therapy approach to help patients with a devastating orphan disease

The venture capital fund AmorChem II is very proud to announce the financing of a new university project focusing on preclinical development of retinal gene augmentation to treat Peroxisome Biogenesis Disorders in the Zellweger spectrum (PBD-ZSD). The fund's financing will bring together three major research groups from the Research Institute of the McGill University Hospital Centre ("RI-MUHC"), the University of Pennsylvania, and the University of Southern California.

"This program is focused on developing and testing a gene therapy construct that may ultimately improve the well-being of patients with a disabling disorder by treating retinal degeneration. Retinal degeneration leading to blindness is a major, untreatable feature of PBD-ZSD. In fact, visual improvement is a critical symptomatic target that can substantially improve quality of life of patients. The collaborators propose to test the gene therapy targeting retinal photoreceptor cells in PEX1 animal models to study recovery of peroxisomal function," says Inès Holzbaur, Managing Partner at AmorChem.

"It is particularly rewarding for us to finance such a promising and impactful program in an indication where the current standard of care is strictly supportive. This project allows AmorChem to address a major need in this multisystem disorder and enable the improvement of communication, learning, mobility and autonomy of patients with PBD-ZSD. In addition, the strategy of using retinal gene therapy could eventually open the door to using gene augmentation in other organ systems affected in this disorder," adds Elizabeth Douville, Managing Partner at AmorChem.

Three seasoned researchers are contributing a wide breadth of experience and knowledge to this project. The collaboration is led by Dr. Nancy Braverman from the RI-MUHC, internationally recognized for her work in peroxisomal diseases. In addition, the collaboration will benefit from the materials generated by Dr. Jean Bennett at the Center of Advanced Retinal and Ophthalmic Therapeutics at the University of Pennsylvania and the mammalian cell technology expertise of Dr. Joseph G Hacia from the University of Southern California.

"The innovative work by Dr. Braverman has tremendous potential to make a difference in the lives of patients with peroxisomal disorders. The partnership between the RI-MUHC, University of Pennsylvania, University of Southern California and Amorchem is an exemplary demonstration of the synergies required for translating this scientific discovery to tangible benefits for patients," adds Bruce Mazer, MD Executive Director and CSO (Interim), Research Institute of the McGill University Health Centre.

If the initial proof of concept studies are viable, AmorChem will exercise its Option to negotiate an exclusive license to the underlying technology.

About AmorChem

AmorChem (www.amorchem.com) is a leading early stage venture capital fund launched in 2011 in Montreal. The AmorChem team utilizes its deep understanding of fundamental science to uncover its therapeutic potential and focuses its core expertise in translational research to accelerate therapeutic drug discovery and development across a broad spectrum of disease areas. The fund capitalises on both its venture capital expertise and its entrepreneurial experience to spark the creation of start-up companies and help shape them into the next generation of biotech companies. With over $85M under management, AmorChem has financed over 30 university projects and started up several biotechnology companies from the fruits of this innovative research.