Overview

A biotechnology company engaged in developing multiple gene therapy candidates across various therapeutic areas - including Central Nervous System (CNS), rare diseases, hereditary, inflammation, oncology, and addiction - partnered with G&L Healthcare Advisors to provide comprehensive regulatory support.

The client required assistance with Phase I and II programs, particularly with regulatory submissions and securing designations that would accelerate their clinical development.

The client sought long-term, strategic regulatory guidance to navigate the complex requirements of developing novel gene therapies, from authoring Investigational New Drug (IND) applications to obtaining Orphan Drug Designation (ODD) and Breakthrough Therapy Designation (BTD).

Challenges

The client faced several regulatory and logistical challenges while developing the products:
Multiple therapeutic areas: The company was developing gene therapy products in multiple therapeutic areas, each presenting unique regulatory hurdles, including varying requirements for non-clinical, clinical, and CMC sections of the IND.
Accelerating development timelines: The client needed strategic support to accelerate the development timeline by securing ODD and BTD from the U.S. Food and Drug Administration (FDA), which required extensive and well-organized submissions.
eCTD publishing capabilities: The project demanded comprehensive electronic Common Technical Document (eCTD) publishing capabilities to ensure timely and compliant submissions to the FDA’s Electronic Submission Gateway (ESG).

G&L’s Approach and Solution

Within this engagement, G&L provided a multi-disciplinary team to support the client’s Phase I and II regulatory submissions across a diverse range of therapeutic areas, including:
IND authoring and submission: G&L authored the non-clinical, clinical, and CMC sections of the IND for multiple gene therapy products, ensuring each submission met the specific regulatory requirements for gene therapies. The team provided detailed guidance on aligning the IND with FDA expectations to support the early clinical phases of development.
Regulatory designation strategy: G&L developed and executed a strategy to secure Orphan Drug Designation (ODD) and Breakthrough Therapy Designation (BTD) for the gene therapy products. This involved carefully crafting the applications to demonstrate the unmet medical need and the potential for significant improvement over existing therapies, key criteria for these designations.
eCTD Publishing and Submission: Leveraging G&L’s eCTD publishing expertise and ESG Gateway capabilities, the team ensured that all submissions were electronically filed in compliance with FDA requirements, facilitating a smooth submission process and ensuring regulatory timelines were met.

Impact and Outcomes

The collaboration between G&L and the client resulted in several positive outcomes:
Broad gene therapy expertise: G&L’s work showcased their expertise in gene therapy regulatory affairs, including CMC, medical writing, and the development of strategic approaches for regulatory submissions. The collaboration spanned multiple therapeutic areas, highlighting G&L’s ability to navigate complex regulatory landscapes for CNS, rare diseases, and oncology, among others.
Securing regulatory designations: The client successfully obtained OD and BT Designations, which significantly accelerated the development timeline for their gene therapy candidates and provided access to FDA incentives such as priority review and market exclusivity.
Long-term partnership: Due to the success of this initial engagement, the partnership evolved into a long-term collaboration, with G&L continuing to support the client’s entire gene therapy portfolio for over two years.