Hubble Therapeutics Chooses Andelyn Biosciences to Produce AAV for LCA16 Treatment

COLUMBUS, Ohio, Oct. 9, 2024 – Andelyn Biosciences, Inc., a prominent and patient-centered cell and gene therapy CDMO, has been chosen by Hubble Therapeutics, LLC, to produce adeno-associated virus (AAV) utilizing its suspension AAV Curator™ Platform to create clinical-grade AAV for treating Leber Congenital Amaurosis 16 (LCA16).

LCA16, an early-onset severe retinal dystrophy, stems from mutations in the KCNJ13 gene, which is vital for an ion channel in retinal pigment epithelium cells. Children with LCA16 experience visual issues within their first five years, progressing to retinal degeneration and blindness over the next two decades. HUB-101, the therapy targeting LCA16, has received FDA's Rare Pediatric Disease and Orphan Disease Designations. Andelyn will enhance and scale-up the HUB-101 process, generate GMP-grade plasmids, and produce GMP vectors containing normal copies of the KCNJ13 gene. This collaboration will enable Hubble Therapeutics LLC (HubbleTx) to conduct Phase 1/2 clinical trials aimed at proving the treatment's safety and effectiveness in adults.

Jeff Sabados, President of Hubble Therapeutics LLC, stated, "Over the past 18 months, we have selected top partners, including Andelyn Biosciences, to create a gene therapy program for adults and children with LCA16." He further expressed gratitude for Andelyn's commitment to supporting Hubble Therapeutics' mission to aid families impacted by this genetic disorder.

Matt Niloff, Chief Commercial Officer at Andelyn Biosciences, remarked, "We are honored to produce the clinical-grade material for HUB-101 and play a crucial role in Hubble Therapeutics' journey to demonstrate clinical benefits for the LCA16 program. Our role as a leading CDMO is accelerating various programs like HUB-101, which offers genuine hope to patients with debilitating diseases."

Andelyn's exceptional quality and scalable development and manufacturing capabilities are spread across three facilities in and near Columbus, Ohio. The company continues to partner with innovative organizations like Hubble Therapeutics to develop transformative gene therapies for various diseases.

Andelyn Biosciences, Inc. is a comprehensive cell and gene therapy CDMO specializing in the development, characterization, and production of viral vectors for gene therapy. With over two decades of experience, Andelyn has produced cGMP material for more than 450 clinical batches and 75 global clinical trials. Operating from three locations in Columbus, Ohio, Andelyn assists 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 accelerates programs and delivers high-quality products by developing and manufacturing processes on its configurable, data-driven AAV Curator™ Platform or tech transfer in an established client program. Their capabilities include cGMP manufacturing for both adherent and suspension processes up to a 2,000-liter capacity. A comprehensive quality system, regulatory support, and supply chain vertical integration further help clients bring their crucial therapies to market.

Hubble Therapeutics LLC (HubbleTx) is a biotechnology company dedicated to patient-focused research, stemming from Professor Bikash Pattnaik's lab at the University of Wisconsin-Madison. The company focuses on developing a novel gene therapy for Leber Congenital Amaurosis Type 16 (LCA16), a severe retinal disease causing significant vision loss in both adults and children. HubbleTx's mission is to restore vision and enhance the quality of life for affected individuals through gene delivery technology targeting the disease's underlying genetic mutation. The company comprises leading scientists, industry experts, and top-tier partners committed to rigorous research and clinical development to ensure that their therapies are safe, effective, and accessible. Through collaboration and innovation, HubbleTx aims to bring hope to those affected by LCA16, striving to create a brighter future for patients battling the condition.