The underwriters for the offering are Leerink Partners, Evercore ISI and Canaccord Genuity.
This is a clinical-stage biotechnology company discovering and developing novel therapies for mitochondrial and neurodegenerative diseases of the eye and, in the future, of the central nervous system. To address these therapeutic areas, Gensight leverages its integrated development platform by combining a gene therapy-based approach with core technology platforms of mitochondrial targeting sequence (MTS) and optogenetics.
In the filing the company detailed:
Our initial focus has been on developing therapies for severe retinal diseases, with the goal of preserving or restoring vision in patients suffering from such diseases. Using our gene-therapy based approach, our product candidates are designed to be administered in a single treatment to each eye by intravitreal or subretinal injection in order to provide patients with a long-lasting functional cure, potentially for the rest of their lives.
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It is also worth noting that Gensight’s pipeline currently consists of two lead product candidates for the treatment of sight-threatening retinal degenerative diseases:
- GS010 as a treatment for LHON due to the ND4 gene mutation, a rare mitochondrial genetic disease. GS010 is based on the MTS technology platform, which permits missing mitochondrial proteins to be shuttled into the mitochondrion, enabling the restoration of mitochondrial function. There is currently no FDA-approved treatment to prevent loss of sight or restore vision in LHON patients.
- GS030 is based on optogenetics, a technology that makes cells responsive to light. Gensight is using a modified AAV2, to which it has exclusive rights in optogenetics, to introduce a DNA sequence that encodes a photosensitive protein, ChrimsonR, into the nucleus of target cells.
The company plans to use the proceeds from this offering to further support the clinical development of GS010 and GS030, with the remainder to fund internal research and development.
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