RARE Daily

Ocugen Raises $23 Million to Advance Ocular Gene Therapies

February 8, 2021

Rare Daily Staff

Ocugen, said it has entered into definitive agreements with healthcare-focused institutional investors for the sale of an aggregate of 3 million shares of its common stock at $7.65 per share in a registered direct offering.

The offering is a premium to its closing price of $5.25 ahead of the announcement.

Ocugen is focused on discovering, developing, and commercializing gene therapies to cure blindness diseases, and to developing a vaccine to fight COVID-19. The company says its modifier gene therapy platform has the potential to treat multiple retinal diseases with one drug – “one to many.”

The technology is licensed from the Schepens Eye Research Institute of Massachusetts Eye and Ear (Harvard Medical School) and involves targeted delivery and expression of one or more nuclear hormone receptor genes (NHRs) in the disease tissues. NHRs play a vital role in regulating retinal cell development, maturation, metabolism, visual cycle function, survival, and maintaining the cellular and molecular homeostatic various tissues, including the retina. Multiple animal models have shown that expression of NHRs within the retina can stabilize retinal cells and suppress/prevent the development of inherited diseases, demonstrating their modifier function.

Ocugen’s OCU400 (AAV-NR2E3) is a preclinical gene therapy product candidate with the potential to be broadly effective in restoring retinal integrity and function across a range of genetically diverse inherited retinal diseases. It consists of a functional copy of a nuclear hormone receptor (“NHR”) gene, NR2E3, delivered to target cells in the retina using an adeno-associated viral (“AAV”) vector.  As a potent modifier gene, expression of NR2E3 within the retina may help reset retinal homeostasis, potentially stabilizing cells and rescuing photoreceptor degeneration.

In five unique mouse models of retinitis pigmentosa (RP), treatment with the AAV-NR2E3 gene by subretinal injection to prevent the further development of multiple genetically diverse inherited retinal diseases by protecting photoreceptors from further damage after disease onset. The five RP models Ocugen has tested include PDE6β associated RP, two different Rhodopsin mutation-associated RPs, Leber congenital amaurosis, and enhanced S-cone syndrome.


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