Hubble Therapeutics Secures $7.3M Series A Funding for Gene Therapy Targeting Rare Childhood Blindness
核心洞察
Hubble Therapeutics (搜索) has raised $7.3 million in Series A funding to advance HUB-101, a gene therapy targeting Leber Congenital Amaurosis 16 (搜索) (LCA16 (搜索)) and Snowflake Vitreoretinal Degeneration (搜索), into clinical trials by 2026.
Andelyn Biosciences (搜索) has been selected to manufacture the adeno-associated virus (搜索) (AAV (搜索)) therapy, which aims to deliver normal copies of the KCNJ13 (搜索) gene to address the root cause of these rare retinal disorders.
HUB-101 has received both Rare Pediatric Disease Designation and Orphan Disease Designation from the FDA, positioning it as a potential breakthrough for children who typically develop visual abnormalities before age five and progress to blindness.
Hubble Therapeutics (搜索) LLC, a patient-focused biotechnology company, has successfully closed a $7.3 million Series A funding round to advance its lead gene therapy candidate, HUB-101, into human clinical trials. The company has simultaneously announced a manufacturing partnership with Andelyn Biosciences (搜索) to produce clinical-grade adeno-associated virus (搜索) (AAV (搜索)) vectors for the treatment of rare pediatric blindness disorders.
HUB-101 targets Leber Congenital Amaurosis 16 (搜索) (LCA16 (搜索)) and Snowflake Vitreoretinal Degeneration (搜索) (SVD (搜索)), two severe early-onset retinal dystrophies caused by mutations in the KCNJ13 (搜索) gene. This gene expresses a critical ion channel in the retinal pigment epithelium cells, and mutations typically manifest as visual abnormalities in children before age five, followed by progressive retinal degeneration over the next two decades, ultimately resulting in blindness.
Manufacturing Partnership Established
Andelyn Biosciences (搜索), a leading cell and gene therapy Contract Development and Manufacturing Organization (CDMO), will utilize its suspension AAV (搜索) Curator™ Platform to manufacture clinical-grade AAV for Hubble's therapy. The CDMO will optimize and scale up the HUB-101 process, generate GMP-grade plasmids, and produce GMP vectors that include normal copies of the KCNJ13 (搜索) gene.
"Over the past 18 months we have identified best-in-class partners including Andelyn Biosciences (搜索) to establish a gene therapy development program for adult and pediatric patients suffering from Leber Congenital Amaurosis 16 (搜索)," said Jeff Sabados, President of Hubble Therapeutics (搜索) LLC. "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."
Matt Niloff, Chief Commercial Officer at Andelyn Biosciences (搜索), expressed enthusiasm about the partnership: "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 (搜索) to demonstrate clinical benefit for the LCA16 (搜索) program."
Scientific Foundation and Regulatory Progress
The gene therapy was 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 (搜索) (WARF (搜索)). HUB-101 has already received both Rare Pediatric Disease Designation and Orphan Disease Designation from the FDA.
"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," said Dr. Pattnaik, scientific co-founder of Hubble Therapeutics (搜索). "The development of the HUB-101 gene therapy for LCA16 (搜索) represents not just the scientific breakthrough, but hope for patients and families affected by such devastating conditions caused by defective ion channels."
Clinical Development Timeline
With the new funding and manufacturing partnership in place, Hubble Therapeutics (搜索) has established a clear pathway toward clinical development. The company has partnered with Virscio (搜索) in New Haven, Connecticut, to conduct IND-enabling studies and expects to begin Phase I/II clinical trials in 2026 to demonstrate the safety and efficacy of HUB-101.
Greg Keenan, Senior Director of WARF (搜索) Ventures & Accelerator and Hubble Therapeutics (搜索) Board of Director, highlighted the significance of the academic-industry partnership: "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."
Addressing an Unmet Medical Need
LCA16 (搜索) and SVD (搜索) represent significant unmet medical needs in ophthalmology. Currently, no approved treatments exist that address the underlying genetic cause of these conditions. By delivering functional copies of the KCNJ13 (搜索) gene, HUB-101 aims to restore the critical ion channel function in the retina and potentially halt or reverse disease progression.
The gene therapy approach is particularly promising for pediatric patients, who may benefit from early intervention before significant retinal degeneration has occurred. If successful in clinical trials, HUB-101 could potentially prevent the progression to blindness that currently affects patients with these rare genetic disorders.
"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 (搜索) and SVD (搜索)," said Sabados. "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."