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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: [email protected] Logo - WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

COLUMBUS, Ohio, Oct. 9, 2024 /PRNewswire/ -- Andelyn Biosciences, Inc., a leading and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), has been selected by Hubble Therapeutics, LLC, to manufacture adeno-­associated virus (AAV) using its suspension AAV Curator™ Platform to manufacture clinical grade AAV for the treatment of Leber Congenital Amaurosis 16 ("LCA16"). LCA16 is a severe, early-onset retinal dystrophy resulting from mutations in the KCNJ13 gene, which expresses a critical ion channel in the retinal pigment epithelium cells in the retina. Children typically present with visual abnormalities within five years after birth, followed by progressive retinal degeneration during the next two decades of life, ultimately resulting in blindness. HUB-101 has received Rare Pediatric Disease Designation and Orphan Disease Designation from the FDA for this program. Andelyn will optimize and scale-up the HUB-101 process, generate GMP-grade plasmids, and produce GMP vectors that include normal copies of the KCNJ13 gene. From this partnership, Hubble Therapeutics LLC ("HubbleTx") will be able to conduct Phase 1/2 clinical trials with the intention to demonstrate safety and efficacy in an adult patient population. "Over the past 18 months we have identified best-in-class partners including Andelyn Biosciences to establish a gene therapy development program for adult and pediatric patients suffering from Leber Congenital Amaurosis 16 (LCA16)," said Jeff Sabados, President of Hubble Therapeutics LLC. Sabados continued, "Andelyn Biosciences has become a global leader in gene therapy development for ultra-rare and orphan diseases and we are grateful for their unwavering commitment toward helping Hubble Therapeutics realize our steadfast promise to support families throughout the world impacted by this devastating genetic disease." Matt Niloff, Chief Commercial Officer at Andelyn Biosciences said, "We are extremely grateful for the opportunity to produce the clinical grade material for HUB-101 and be a critical part of the pathway for Hubble Therapeutics to demonstrate clinical benefit for the LCA16 program. We are thrilled that our establishment as a top clinical and commercial CDMO is helping to accelerate so many programs like HUB-101 which offer real hope to patients stricken with debilitating diseases." With exceptional quality and scalable end-to-end development and manufacturing capabilities across its three facilities located in and near Columbus, Ohio, Andelyn continues to partner with premier innovator organizations like Hubble Therapeutics to enable the development of life-altering gene therapies for rare and prevalent diseases. About Andelyn Biosciences, Inc. Andelyn Biosciences is a full-service cell and gene therapy CDMO focused on the development, characterization and production of viral vectors for gene therapy. With more than 20 years of experience, Andelyn's deep scientific expertise has resulted in the production of cGMP material for more than 450 clinical batches and 75 global clinical trials. Operating out of three Columbus, Ohio facilities, Andelyn supports its clients in developing cell and gene therapies from concept through plasmid engineering and manufacturing, process and analytical development, and cGMP clinical and commercial manufacturing. Andelyn can accelerate programs and deliver high quality products by developing and manufacturing processes on its configurable, data-driven AAV Curator™ Platform, or tech transfer in an established client program. Capabilities include cGMP manufacturing for both adherent and suspension processes up to a 2,000-liter capacity. A rigorous quality system, regulatory support and supply chain vertical integration further support clients in bringing their critical therapies to market. For more information, visit andelynbio.com. About Hubble Therapeutics, LLC Hubble Therapeutics LLC ("HubbleTx") is a patient-focused biotechnology company developing research out of Professor Bikash Pattnaik's lab at University of Wisconsin-Madison. Our primary focus is on developing a novel gene therapy for Leber Congenital Amaurosis Type 16 (LCA16), a debilitating retinal disease that leads 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. Through collaboration and innovation, HubbleTx strives to bring hope to those affected by this condition. Together, we aim to illuminate the path to a brighter future for patients battling LCA16. SOURCE Andelyn Biosciences WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED