The Center for Breakthrough Medicines (CBM), a contract development, manufacturing, and testing organization (CDMO) is packaging their preclinical capabilities into an IND-enabling offering to help innovator companies manufacture high quality drug substance and drug product for preclinical studies needed for IND-enabling activities.

Preclinical assessment of investigational cell and gene therapy products is a critical milestone to move programs forward while balancing limited capital allocated to this R&D stage. The importance of high-quality preclinical material for IND-enabling studies is often underestimated, leading to programs not having the data required to get their IND accepted and limited cash and resources to redo studies.

CBM’s IND-enabling preclinical package offers support for preclinical studies and includes off-the-shelf AAV rep-cap and helper plasmids, custom plasmid manufacturing, access to a proprietary cell line, streamlined process development services, scalable preclinical vector manufacturing services, as well as regulatory support for INTERACT and Pre-IND meetings.

According to the company, the package enables therapeutic developers to generate high quality material and data in under five months and positions them to move rapidly towards IND approval and GMP clinical manufacturing.

CBM’s preclinical offerings will supply drug substance and drug product for proof-of-concept, toxicology, safety, biodistribution studies, method development, interim reference standard, development stability, and drug product development critical for IND packages. Additionally, these productions utilize similar processing techniques as would be used for GMP manufacturing, eliminating comparability risks associated with process changes. 

Additional analytical testing and regulatory services are available to prepare data briefing packages for INTERACT and Pre-IND meetings. Upon preclinical manufacturing completion, clients have full transparency to the data from execution, production, and purification, a critical data set for the sponsor of any clinical program.

“The past year has been a catalyst moment for gene therapies with approvals for AAV- and LV- based gene therapies driving renewed momentum in the pre-clinical pipeline,” said Avi Nandi, CBM’s chief technical officer. “CBM’s pre-clinical program will help the next generation of gene therapy companies learn from the lessons of the last five years and lay a smoother path through clinical trials and regulatory submission.”