Andelyn Biosciences Selected by Hubble Therapeutics to Manufacture Clinical Grade AAV for the Treatment of Leber Congenital Amaurosis (LCA16)

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