On a mission to treat patients not covered by Luxturna, Horama refuels with a Series B expansion and a new name
21 Jul 2021
Gene TherapyAntibodyOrphan DrugADC
About four years after closing its Series B round the first time, Horama is adding another $25.1 million to its coffers for its work on a next-gen gene therapy that could treat retinitis pigmentosa patients not covered by Roche’s Luxturna — and it’s sporting a new name to boot.
The French biotech, now called Coave Therapeutics, has expanded its Series B round, bringing its total raise to $39 million, CEO Rodolphe Clerval announced early Wednesday morning. The latest round was led by Seroba Life Sciences, with a hand from Théa Open Innovation, eureKARE, Fund+, Omnes Capital, V-Bio Ventures, Kurma Partners, Idinvest, GO Capital, and Sham Innovation Santé/Turenne.
Why the name change? Coave stands for “conjugated AAV vectors,” which is exactly what the company intends to make, Clerval told Endpoints News.
Horama launched in 2015 based on work from the University of Nantes in the west of France. The company’s core technology — dubbed the AAV Ligand Conjugate, or ALIGATER platform — is designed to enhance the delivery and efficiency of AAV vectors by binding AAVs to ligands, similar to the way scientists would link an antibody to a payload to form an antibody-drug conjugate.
“It’s a technology that is inspired from, for instance, antibody drug conjugates, where people are taking chemical components and using chemistry to bind them on proteins,” Clerval said. “This is exactly what we are doing, but we are taking the entire AAV… and we are developing specific ligands and we are binding covalently those ligands onto the surface of the capsid.”
By doing so, Clerval says the scientists are able to make three key changes: improving the biodistribution of the capsid (how it’s able to travel through the tissue of an organ), improving transduction efficiency (its ability to transfer the payload inside the cell), and preventing the capsid from being neutralized by the body’s immune response.
The company’s lead program, CTx-PDE6b, is currently in a Phase I/II trial for PDE6b inherited retinal dystrophies, including retinitis pigmentosa. Luxturna — which was developed by Spark Therapeutics before it was bought out by Roche in 2019 — is currently approved to treat mutations in the RPE65 gene.
Retinitis pigmentosa caused by PDE6b mutations often leads to blindness by midlife, and is characterized by the progressive loss of photoreceptors.
“Those patients basically will lose some photoreceptors, lose their vision, starting by the periphery vision to a shrinkage of their visual field,” Clerval said.
Last August, Ocugen received orphan drug designation for its own gene therapy, OCU400, to treat PDE6b-associated retinal diseasesPDE6b-associated retinal diseases. That candidate’s currently in IND-enabling stages, according to the company’s website.
To date, 15 of 17 patients are enrolled in Coave’s Phase I/II trial, and Clerval expects to read out data in 2022. In addition to advancing that program toward pivotal trials, the 12-person team also has multiple preclinical programs in the works for rare CNS and ocular diseases, including CTx-GBA1 for Parkinson’s disease and Gaucher Disease and CTx-ABCA4 for Stargardt’s disease.
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