Hubble Therapeutics closes Series A Financing to develop gene therapy cure for LCA16

HANOVER, N.H., March 25, 2025 /PRNewswire/ -- Hubble Therapeutics LLC a patient-focused biotechnology company developing a gene therapy to cure pediatric blindness, announces the successful closing of a $7.3 million Series A funding round, which will advance its lead candidate, HUB-101, into human clinical trials.

Hubble Therapeutics' gene therapy, HUB-101, targets two rare and devastating eye disorders: Leber Congenital Amaurosis (LCA16) and Snowflake Vitreoretinal Degeneration (SVD). This innovative treatment, developed by Dr. Bikash Pattnaik at the University of Wisconsin-Madison School of Medicine and Public Health and licensed from the Wisconsin Alumni Research Foundation (WARF), aims to address the root cause of these conditions. LCA16 and SVD are severe, early-onset retinal dystrophies caused by mutations in the KCNJ13 gene, which is crucial for the function of a vital ion channel in the retina's pigment epithelium cells. These disorders typically manifest in children before the age of five, leading to progressive retinal degeneration over the following two decades and ultimately resulting in blindness.

"For over the past 20 years, my lab has developed in vitro and in vivo models of ion channelopathy to develop curative gene therapies for several devastating pediatric blindness diseases," Dr. Bikash Pattnaik, professor in the Departments of Pediatrics, Ophthalmology and Visual Science at the University of Wisconsin–Madison, Daniel M. Albert Chair McPherson Eye Research Institute and scientific co-founder of Hubble Therapeutics LLC, "The development of the HUB-101 gene therapy for LCA16 represents not just the scientific breakthrough, but hope for patients and families affected by such devastating conditions caused by defective ion channels. As we push these therapeutic boundaries, we empower a future where innovative ion channel therapies in our lab address genetic diseases at their core."

To date, HUB-101 has received Rare Pediatric Disease Designation and Orphan Disease Designation from the FDA for this program. It has partnered with Andelyn Biosciences in Columbus, Ohio, and Virscio in New Haven, Connecticut, to develop, manufacture, and conduct IND-enabling studies. Hubble Therapeutics expects to enter the clinic to conduct Phase I / II trials in 2026 to demonstrate the safety and efficacy of HUB-101.

"This groundbreaking research aims to restore vision in patients affected by a rare eye disease that ultimately results in complete blindness, with WARF's investment in Hubble Therapeutics as the Series A co-lead investor, we are thrilled to continue our support for the development of a disease-modifying treatment," says Greg Keenan, Senior Director of WARF Ventures & Accelerator and Hubble Therapeutics Board of Director. "The collaboration between Hubble Therapeutics and the pioneering research of UW-Madison inventor, Dr. Bikash Pattnaik, exemplifies the transformative potential of academic-industry partnerships and we are honored to continue our support in advancing this critical research alongside the dedicated team at Hubble Therapeutics."

"We are grateful for our investors, leading scientists, industry experts, best-in-class partners, patient families, and the University of Wisconsin-Madison who share our commitment to realizing the promise of improving vision for patients suffering from LCA16 and SVD," said Jeff Sabados, Founder and President of Hubble Therapeutics LLC. Sabados continued, "with our new financing and the establishment of key partnerships, we hope to scale up HUB-101 manufacturing and conduct additional IND-enabling studies to enter the clinic within the next two years to realize our steadfast, unwavering promise to support families throughout the world impacted by this devastating genetic disease."

About Hubble Therapeutics, LLC
Hubble Therapeutics LLC ("HubbleTx") is a patient-focused biotechnology company developing research from Professor Bikash Pattnaik's lab at the University of Wisconsin-Madison. Our primary focus is on developing novel gene therapy for Leber Congenital Amaurosis Type 16 (LCA16) and Snowflake Vitreoretinal Degeneration (SVD), two debilitating retinal diseases that lead to severe vision loss in adult and pediatric patients. Our mission is to restore vision and improve the quality of life for affected individuals and their families by harnessing the power of gene delivery technology to target the underlying genetic mutation responsible for the disease. Our team of leading scientists, industry experts, and best-in-class partners are committed to rigorous research and clinical development, ensuring that our therapies are safe, effective, and accessible. HubbleTx strives to bring hope to those affected by this condition through collaboration and innovation. Together, we aim to illuminate the path to a brighter future for patients battling LCA16 and SVD.

For Hubble media inquiries, please contact:
jsabados@hubbletx.com

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