Positive data from Unity Biotechnology Inc.’s phase II study of UBX-1325, a senolytic BCL-XL inhibitor for treating diabetic macular edema, boosted the stock out of the penny range. Shares of the South San Francisco-based company’s stock (NASDAQ:UBX) rose sharply, 54% on Aug. 12, to $1.31 each.
The phase III miss disclosed Aug. 11 by Kubota Pharmaceutical Holdings Co. Ltd. subsidiary Kubota Vision Inc. in Stargardt disease put more eyes on the rare, inherited, juvenile-onset form of macular degeneration, for which nothing is approved.
Researchers are closer to better diagnosing and treating age-related macular degeneration (AMD) after discovering new genetic signatures of the disease by reprogramming stem cells to generate high-resolution disease models.
Frontera Therapeutics Inc. raised $160 million in a series B funding round to develop its lead gene therapy product candidate for retinal disease, FT-001, for which INDs have been approved by the U.S. FDA and China NMPA.
China’s National Medical Products Administration (NMPA) approved Ocumension Therapeutics Ltd.’s and Eyepoint Pharmaceuticals Inc.’s drug-device combination product, Yutiq (fluocinolone acetonide intravitreal implant), for chronic non-infectious uveitis affecting the posterior segment of the eye.
Kriya Therapeutics Inc. has raised a $270 million series C financing to further develop its pipeline of gene therapies for treating cancer, ophthalmological problems, and rare and chronic diseases. The Redwood City, Calif.-based company has greatly expanded its employee roster, from about seven people to around 160 people, since its $80 million series A in May 2020 and scaled its learning-enabled tech and cloud computing abilities. It also further solidified its technology, manufacturing, R&D, and therapeutics units, something it plans to continue with the series C money.
Although gene therapy is now “a clinical reality,” it still remains an early stage therapeutic modality. That’s the view of Caroline Man Xu, CEO and co-founder of Vigeneron GmbH, a German gene therapy company that has maintained a low profile while steadily staking out a promising position in gene therapies for inherited retinal disease.
Visus Therapeutics Inc. has out-licensed phase III candidates Brimochol and Carbachol to Hong Kong’s Zhaoke Ophthalmology Ltd. to develop and commercialize its long-acting, presbyopia-correcting eye drops in greater China, South Korea and select Southeast Asian territories.
Askgene Pharma Inc., which less than two weeks ago reported positive initial data from an ongoing phase I/II trial testing its claudin 18.2-targeting candidate, ASKB-589, added $20 million in a series A round, intended to advance the company’s clinical pipeline and support further development of its Smartkine cytokine drug platform.
Shares in Oxurion NV dropped 40.3% percent May 9 on news that one of its two clinical-stage assets, THR-687, failed to demonstrate efficacy in a phase II trial in diabetic macular edema (DME). The candidate, a small-molecule pan integrin receptor antagonist, failed to demonstrate efficacy in Part A of the trial, called Integral, in which treatment-naïve patients received one of two doses of THR-687.