In collaboration with Alpine BioTherapeutics Corporation, Allele Biotechnology and Pharmaceuticals will make therapies for retinal diseases based on induced pluripotent stem cells (iPSCs)

The deal, financials of which have not been divulged, sees the two firms enter into a research agreement for developing iPSC-derived cell therapy for treating diseases including blindness caused by Retinitis Pigmentosa, Stargardt disease, and dry AMD.

Alpine, which has a patented stem cell differentiation technique used to generate human retinal stem cells from pluripotent stem cells, gains access to Allele’s cGMP facility and will license one or more of Allele’s cGMP-grade human iPSC lines to generate these cell therapy products.

Image: iStock/Dr_Microbe

Allele’s iPSC lines are generated and differentiated using mRNA (messenger RNA) technology.

“We introduce mRNAs to encode genes that will turn adult cells into pluripotent stem cells by a process called transfection,” a spokesperson from Allele told this publication. This involves sending a message to human adult cells telling them to go back to the embryonic stage as fully pluripotent stem cells, without ironing the letters carrying the message permanently into the genome of the cells.

“Because mRNA molecules do not alter the genome of the cells, we call the resulting cells footprint-free,” meaning the stem cells can be generated within a much shorter time period, at a higher efficiency, and with minimum effort, the company explained.

San Diego plant

The work will take place at Allele’s site in San Diego, California, from a facility specifically built to handle stem cells and the cells that are derived from them. Allele acquired the 18,000 square-foot site in 2015.

“Currently Allele Biotech can make dozens, to eventually hundreds, of iPSC lines per year, and concurrently produce tissue specific cells differentiated from iPSCs, such as eye stem cells, pancreatic beta cells, neural progenitor cells, etc,” we were told.

“Since our operations began, Allele Biotech has been using the most efficient protocols in our cleanroom setting which means significant cost reduction.”

The announcement comes two months after Allele inked a deal with SCM Lifescience to develop diabetes therapies using pancreatic beta cells derived from induced pluripotent stem cells (iPSCs).