Investing over $100m in the expansion of its Rockville headquarters, including more than $65m dedicated to this new manufacturing innovation center, the gene therapy developer said it has also hired 200 people over the past two years in its move to establish end-to-end capabilities in gene therapy from research and early development to commercial ready manufacturing,
The GMP facility is located in Regenxbio’s 132,000 square foot (12263.2 sq. m) headquarters in Rockville, Maryland.
The company said the site will enable it to boost manufacturing of its NAV technology-based adeno-associated virus (AAV) vectors at scales up to 2,000 liters, making it one of only a few gene therapy companies worldwide able to do so.
Designed to meet global clinical and commercial regulatory standards, and including two independent bulk drug substance production suites, a final drug product suite and integrated quality control labs, the facility will also implement the company’s NAVXpress platform suspension cell culture process, aimed at improved purity and yield.
“In-house manufacturing is a key differentiator for Regenxbio as a leader in gene therapy,” said Curran Simpson, chief of operations and technology officer at Regenxbio. “Quality manufacturing is crucial to all stages of AAV gene therapy development… bringing our manufacturing in-house allows us to control the process from beginning to end and provides flexibility to support a wide range of clinical and commercial needs.”
Indeed, that control aspect came to the fore in May this year when Regenxbio announced it had to delay dosing of patients in its DMD gene therapy clinical trial following a quality issue at an unnamed third-party contract manufacturer.
The US company’s in-house pipeline targets rare and more common diseases in retinal, metabolic, and neurodegenerative therapeutic areas.
Its NAV technology platform consists of over 100 novel adeno-associated virus (AAV) vectors, one of which was used in the US Food and Drug Administration (FDA) approved gene therapy, ZOLGENSMA, for spinal muscular atrophy in children under two years old. The developer has also licensed out its technology to a growing list of partners and licensees that includes Novartis, Eli Lily and Pfizer.
Last September saw it ink a deal with AbbVie to develop and commercialize its one-time gene therapy treatment for wet age-related macular degeneration, diabetic retinopathy and other chronic retinal diseases.
And it has committed to a ‘5x'25’ strategy to progress five AAV therapeutics from its internal pipeline and licensed programs into pivotal-stage trials or commercial products by 2025.