Armatus Bio’s lead candidate for CMT1A has received orphan drug designation and rare paediatric drug designation from the FDA
Andelyn Biosciences and Armatus Bio strike deal for manufacturing the latter’s candidate for CMT1A. (Credit: Sangharsh Lohakare on Unsplash)
Andelyn Biosciences and biotechnology company Armatus Bio have entered into a manufacturing agreement for the latter’s gene therapy candidate for Charcot-Marie-Tooth Type 1A (CMT1A), a rare neurological disease.
Based in Ohio, Andelyn Biosciences is a contract development and manufacturing organisation (CDMO) focused on cell and gene therapies.
Armatus Bio, which is a privately held biotechnology innovator in the late preclinical stage, utilises vectorized RNAi to target critical unmet medical needs in genetically-driven neurological diseases.
Under the terms of the partnership, Armatus Bio will use Andelyn Biosciences’ experience in producing adeno-associated viruses (AAVs). The former will also use the CDMO’s proprietary suspension platform for plasmid synthesis, viral vector toxicity, GMP clinical manufacturing, and development operations.
Armatus Bio’s lead candidate for CMT1A has received orphan drug designation and rare paediatric drug designation from the US Food and Drug Administration (FDA).
Currently, the asset is progressing towards investigational new drug (IND)-enabling studies.
Andelyn Biosciences chief commercial officer Matt Niloff said: “We are extremely moved by Armatus Bio’s commitment to address the urgent unmet needs in CMT1A and are impressed by its innovative therapeutic technology for the disease.
“We look forward to scaling up the process in our program-specific, configurable suspension platform, and accelerating the therapy into the clinic with the highest degree of quality.”
Andelyn Biosciences offers development, plasmid production, and manufacturing services at its three locations in Columbus, Ohio.
The CDMO aims to advance customer discoveries and produce gene and cell therapies for both common and uncommon diseases.
The company has a cGMP manufacturing capacity for both adherent and suspension processes up to a 2,000-litre capacity.
Armatus Bio chief technical officer Brian Price said: “Andelyn has demonstrated its strong capabilities in manufacturing complex genetic medicines with high quality and consistency, which will be critical to accelerating our development efforts.
“We look forward to collaborating with Andelyn on this program as we work toward supporting this population that urgently awaits innovative solutions.”