Novartis Adds to Gene Therapy R&D Portfolio


A new deal has been signed between Novartis and Homology Medicines as Novartis continues to drive its research and development work in cell and gene therapy. Homology will give Novartis the use its gene editing technology for the development of novel treatments for ophthalmic and hemoglobinopathy diseases, although it is unknown exactly what the companies are seeking to target.

While exact financials have not been disclosed, Homology will receive a direct payment in addition to an equity investment to be used “to advance the programs and to explore new opportunities for Homology’s technology platform”. The agreement retains Homology’s U.S. commercial rights and it arranges profits to be shared with Novartis for in vivo applications related to the hemoglobinopathy program. This technology adds to what Novartis has in its phoenix-from-the-flames cell and gene therapy pipelines, which includes CAR-T and collaborative work on CRISPR-Cas9.

Lloyd Klickstein, M.D., Ph.D., head of translational medicines at Novartis, stated in an interview, “With this collaboration, Novartis is adding another emerging technology to our cell and gene therapy toolbox. We believe Homology’s technology has great potential for development of new therapies”.

Klickstein added, “Their technology, known as AAV-mediated editing by directed homologous recombination (AMEnDR), uses adeno-associated virus (AAV) vectors isolated from human CD34-positive cells for genome editing. Uniquely, Homology’s AAVs are able to trigger homologous recombination, a naturally occurring process, to change DNA letters inside cells and correct specific gene mutations with what appears to be efficiency greater than that observed with other gene editing technologies being explored today”.

Klickstein believes that this agreement is part of the company’s strategic drive to “work with innovative leaders” in the developing genome editing field. Presently, Homology’s leading candidate is in preclinical IND-enabling trials and has been scheduled for upcoming trials in an inborn error of metabolism disease.


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