Combined gene therapy expertise supports US biotech
The client was an early-stage start-up in the pre-clinical stage of developing a suite of gene therapy products for a cardiovascular indication. The client had extensive pre-clinical data, but as a small team with no pharmaceutical development experience, they requested Boyds to provide a development and regulatory roadmap to IND/CTA and help to identify a short list of potential CDMOs to manufacture the product and provide all the necessary CMC activities for the program.
The project was undertaken collaboratively between the Product Development and Regulatory teams at Boyds and involved a detailed interrogation of the data packages and documentation made available by the Client.
As the specific clinical indication and target patient population were yet to be identified, the regulatory team provided a generic gene therapy regulatory roadmap based on Boyds’ considerable experience in this field, outlining the following:
- Current US and UK/EU regulatory guidance for the development of gene therapy products,
- Competitive landscape and regulatory precedence,
- US and European regulatory requirements for a first-in-human (FIH) clinical study,
- Overview of GMO/biosafety applications and requirements,
- Opportunities for early interaction with FDA and EMA, and recommendations on questions/topics for discussion,
- Overview of product designations that could be pursued and timings for consideration.
The product development team at Boyds:
- Managed the programme both internally, liaising with regulatory colleagues and other specialist consultants, and externally as a point-of-contact for the Client,
- Provided considerations for the nonclinical plan, in line with current regulatory guidance,
- Provided CMC considerations and a short list of CDMOs,
- Provided a high-level overview of an integrated development roadmap (CMC, nonclinical and regulatory) through to IND application/CTA submission.
Impact and Outcomes
This cross-functional output provided by the Boyds team will enable the Client to create a detailed plan to IND/CTA which combined with the key milestones identified by the Boyds team, will allow the Client to provide potential investors with confidence that they can execute a program leading to opening an IND/a successful CTA(s).