Biogen plans to build a new gene therapy manufacturing facility
in Research Triangle Park
Biogen, a pioneer in neurosciences, plans to build a new gene therapy manufacturing facility at its Research Triangle Park (RTP) manufacturing campuses in North Carolina to support its growing gene therapy pipeline across multiple therapeutic areas. As pioneers in neuroscience, Biogen is pursuing multiple modalities with the aim of bringing life-saving medicines to those who suffer from serious neurological and neurodegenerative diseases. With this new facility, Biogen is investing in robust and scalable gene therapy manufacturing with the goal of ensuring reliable supply to patients worldwide. “We plan to build differentiated, sustainable and advanced manufacturing capabilities to support our gene therapy programs and collaborations,” said Nicole Murphy, Senior Vice President, Global Manufacturing and Technical Operations. “This additional investment underscores our commitment to RTP and our mission to deliver a reliable supply of high-quality medicines to the patients we serve. We are poised with a diverse workforce that is passionate about making a difference in the lives of patients and we look forward to welcoming new colleagues from the community to join us in that mission.”
Through the new facility, Biogen aims to continue its investment in North Carolina and expand its existing operations and combined workforce of approximately 1,900 employees at both campuses in RTP. Biogen anticipates that the new facility will create approximately 90 new jobs, with an estimated total investment of approximately $200 million. Biogen selected RTP for this new facility given the region’s diverse pool of talent and the company’s 26-year track record of attracting highly qualified and passionate employees in North Carolina. The site will be 175,000 square feet and designed in compliance with the most advanced sustainability standards regarding energy use, waste management and water consumption. Currently, Biogen is developping two AAV-based gene therapy products for inherited retinal diseases:
•BIIB111 (timrepigene empavorvec – AAV2 containing the human REP1 gene)), which is in phase III for the treatment of choroideremia (data expected by H1 2021)
•BIIB112(RPGR gene therapy – AAV8 containing a codon-optimized RPGR gene), which is in phase II-III for the treatment of X-linked retinis pigmentosa (data expected by H1 2021).
These assets have been developed by Nighstar Therapeutics which was acquired by Biogen in June 2019.
Biogen’s development pipeline also includes four antisense oligonucleotides (ASO):
•BIIB067 (tofersen) for the treatment of superoxide dismutate 1 (SOD1) amyotrophic lateral sclerosis (ALS). In July 2020 final positive results from a phase I/II study were published in the New England Journal of Medicine. These results demonstrated proof-of-concept and proof-of-biology of tofersen, which is currently being investigated in the ongoing Phase 3 VALOR study. Phase 3 data are now expected by H2 2021.
•BIIB105 (ASO targeting ataxin-2) in ALS. Last September the first patient was dosed in a phase 1 study that evaluates safety, tolerability and pharmacokinetic profile of BIIB105 in participants with ALS) or poly-CAG expansion (polyQ)-ALS.
•BIIB094 (ASO designed to bind to the Leucine Rich Repeat Kinase 2 (LRRK2) mRNA and mediate its degradation, reducing LRRK2 protein levels) for the treatment of Parkinson’s disease. This ASO is developed in collaboration with Ionis Pharmaceuticals and a phase 1 is ongoing.
•BIIB101 (ASO designed to bind to inhibit the production of the alpha-synuclein protein) as a potential therapy for Parkinson’s disease, multiple system atrophy, and related synucleinopathies. This ASO is also developed in collaboration with Ionis Pharmaceuticals. In July 2020, Biogen initiated a phase I/II study evaluating ION464 in patients with multiple system atrophy.
Anne-Lise Berthier (07/03/2021=