Fresh from selling Trophos to Roche, Placet bags cash for gene therapy upstart

(By Benh LIEU SONG - Own work, CC BY-SA 3.0,

EuroBiotech logoChristine Placet has rounded up money for her first venture since leading Trophos to a $470 million ($521 million) takeover by Roche ($RHHBY). The financing sees Placet secure Series A funding to bankroll her plans for Horama, an ophthalmic gene therapy company that is advancing a treatment for retinitis pigmentosa toward the clinic.

Paris, France-based Horama set up shop in 2014 to develop the work of a scientific founding team that was among the first to trial an ophthalmic gene therapy in humans in France. While this background and the support of gene therapy leaders in Montpellier and Nantes put Horama in a strong position, it has kept a low profile over the past couple of years, over which period rivals such as its compatriot GenSight Biologics have made headlines on both sides of the Atlantic.

Now, Horama is ready to step up its activities. After spending a decade at Trophos that culminated in Roche swooping in for its spinal muscular atrophy program, Placet joined Horama as CEO earlier this year. Placet put some of her own money into the company, and has persuaded Omnes Capital, Sham Innovation Santé and GO Capital to write checks. The syndicate of French VCs has invested €4 million in Horama, a sum the company will use to take its treatment for retinitis pigmentosa into Phase I in the first quarter of next year.

The retinitis pigmentosa treatment, HORA-PDE6B, is designed to correct a defect in the PDE6B gene. Horama plans to inject the suspension of recombinant adeno-associated virus (rAAV) vectors into the subretinal space of people who have the defective gene. Once in situ, Horama expects the viral capsids will deliver non-mutated copies of PDE6B into the photoreceptor rod cells that are vital for vision. The end goal is for these rods to express functional PDE6ß protein, preventing the retinal degeneration that afflicts people with defective forms of the gene.

Horama has already tested the rAAV technology in the clinic, although this lead candidate, a gene therapy against Leber congenital amaurosis type 2, is not the focus of the Series A. HORA-PDE6B is the top priority. Horama is also planning to invest in another preclinical program, HORA-RLPB1. This gene therapy is designed to deliver the functioning RLBP1 gene that is lacking in patients with retinitis punctata albescens, another hereditary eye condition. Horama’s pipeline also features a preclinical program against choroideremia.

The focus on the eye means Horama is aiming its gene therapies at an enclosed, easy-to-access organ, characteristics that simplify administration and limit the risk of side effects. While this is a boon for Horama, it is far from the only company to recognize the benefits. Fellow Parisian biotech GenSight is developing ophthalmic gene therapies, as are the U.K.’s Athena Vision and NightstaRx. Despite strong interest in the sector, the large number of genetic faults that affect vision mean there are opportunities to go after conditions where competition is currently minimal or nonexistent.

- read the release (PDF)