Sofinnova Invests in Three New Rare Disease Gene Therapy Startups

European venture capital firm Sofinnova Partners announced three new investments in startups from the Sofinnova Telethon Fund, its early-stage fund dedicated to investments in rare, genetic diseases: AAVantgarde Bio, Alia Therapeutics, and Borea Therapeutics.

Photo: Paola Pozzi, partner at Sofinnova Partners

The Sofinnova Telethon Fund is the largest fund entirely dedicated to biotechnology in Italy. The three new seed investments, totaling $7.1 million (€6 million), align with the fund’s investment strategy to seek out and support the best Italian science and the most promising entrepreneurs to develop world-class companies in the field of rare, genetic diseases.

AAVantgarde Bio is developing gene therapies for inherited retinal disorders. The founder, Alberto Auricchio, is a recognized scientist and a pioneer in the field of gene therapy. The company’s technology builds on existing AAV, or adeno-associated virus-based platforms, which are the main viral vector technology used in gene therapy applications. Existing AAV-based platforms have been limited by capacity, and AAVantgarde Bio responds to this challenge by enabling delivery of large genes to tissue and cells in vivo. The company is a spin-off of TIGEM, an international research institute based in Naples that is owned and managed by the Telethon Foundation.

Alia Therapeutics is developing next generation CRISPR-based medicine for safer on-target in vivo gene editing approaches to the treatment of retinal degeneration. Alia is a spin-off of the University of Trento and was accelerated at BiovelocITA, Italy’s first biotechnology accelerator co-founded in 2015 by Sofinnova Partners and two serial entrepreneurs, Silvano Spinelli and Gabriella Camboni. It is the second company to emerge from the accelerator. This round of financing also included participation from BiovelocITA, Indaco Venture Partners, and a group of Italian investors brought together by Banor SIM and Banca Profilo.

“Both companies have highly qualified scientific teams with globally recognized expertise in the field of gene therapy and gene editing,” said Paola Pozzi, partner at Sofinnova Partners, board member of AAVantgarde Bio and an investment partner in Alia Therapeutics. “We are confident in the potential of these companies to tackle the limitations of existing therapies and develop treatments to diseases that do not have adequate solutions today.”

Borea Therapeutics is advancing a new generation of gene therapies that would enable targeting of specific tissues and cells. The company’s technology could enable viral vectors to reach areas like the central nervous system and peripheral nervous system, thus opening the door to new therapeutic options. Borea Therapeutics was co-founded by Professor Paul Heppenstall and the European Molecular Biology Lab (EMBL), an intergovernmental research organization with six research sites throughout Europe, including one in Rome. The company’s research activities are performed in collaboration with Scuola Internazionale Superiore di Studi Avanzati (SISSA), a scientific center of excellence in Trieste.

“One of the major limitations of gene therapy is the difficulty of selectively targeting specific cells or tissues,” said Lucia Faccio, Partner at Sofinnova Partners and a board member of Borea Therapeutics. “Borea Therapeutics’ technology has the potential to pinpoint specific tissues and cells, which would enable the use of lower doses, making treatment safer and more effective for patients. This could be the next breakthrough in gene therapy.”

Author: Rare Daily Staff