Q&A: Shifting the Biopharma Paradigm from Discovery to Design

Explain what you mean when you say biopharmaceutical innovation must shift from discovery to design?

 Advances in genomics, proteomics and other molecular sciences are enabling researchers to move beyond the traditional method of discovery, which is a laborious process of screening hundreds of molecules against biological targets. Instead, they can embrace “programmable biology,” using rapidly expanding multi-omics knowledge to design therapies that precisely target genetic mutations, mRNA abnormalities, protein dysfunction and dysregulated cellular pathways. This integrated approach has already led to groundbreaking modalities such as CRISPR gene editing, antisense nucleotides and CAR-T cell therapies. As we continue to gain deeper insights into how human biology can be programmed to fight diseases, even more transformative innovations will follow.

 What are the major hurdles the industry must overcome to successfully make this shift?

 Developers of genomic medicines are struggling to translate discoveries into therapies in ways that are both efficient and scalable—and that can reach patients quickly. For example, gene editing technologies have demonstrated their curative potential in the clinic, yet their widespread development remains slow. That’s because they’re highly individualized and costly to develop. Therefore, their potential impact has been limited, particularly for rare diseases. Without a fundamental shift in how gene-editing therapies are developed, the task of bringing them to market will remain prohibitively expensive and unsustainable.

 What changes should be made in the discovery process to enable faster, more efficient therapy design?

 Developers have the opportunity to embrace a platform-based approach to designing genomic medicines. Here’s what I mean by that: the main components of a gene editing therapy are an enzyme, a guide RNA and a delivery system. For many diseases, the enzyme and the delivery system can be reutilized to treat the same cell and tissue, and the only change required is the guide RNA. Therefore, once a therapy targeting a specific genomic mutation in that particular cell or tissue proves effective, the same development framework could be adapted to address different conditions simply by substituting the gRNA.

 This rational, modular design framework will help speed up the development of new therapies and enable them to be precisely tailored to the unique characteristics of each patient’s disease.

 What are the key regulatory processes that need to be revised to support this paradigm shift?

 Under the current regulatory paradigm, adapting a clinically validated therapy for a new disease requires requalification of all components. Recognizing this need, the U.S. Food and Drug Administration introduced the Platform Technology Designation Program for Drug Development Guidance for Industry in 2024. This guidance aims to establish a clear pathway for developers to reuse approved delivery vectors and gene-editing technologies, reducing the need to repeat certain studies.

 There also needs to be a fundamental shift in the industry’s approach to manufacturing. Today, manufacturers must adhere to Good Manufacturing Practice (GMP) regulations, which involve exhaustive, time-consuming and costly qualification processes for each new therapy. These processes are often incompatible with the urgent treatment windows patients face. Ideally, a risk-appropriate manufacturing approach could provide a more efficient and scalable alternative where manufacturers could instead leverage pre-approved enzymes, delivery systems and production platforms. This would streamline manufacturing, significantly reducing time and cost while maintaining safety and quality.

 How is Danaher supporting this transition to a design paradigm?

 To establish new standards for a platform-based approach to gene editing, Danaher and the Innovative Genomics Institute (IGI)—led by UC Berkeley and UC San Francisco—launched in 2024 the Danaher-IGI Beacon for CRISPR Cures. Our goal is to develop scalable platform technologies that will drive the advancement of gene-editing therapies for rare genetic disorders, specifically inborn errors of immunity (IEIs). We believe that the platform approach will transform what once took years into a process that can be accomplished in months.

 To further refine the platform designation process from a regulatory point of view, industry leaders gathered last November for a scientific exchange with the FDA sponsored by Danaher, the Alliance for Regenerative Medicine and the International Society for Cell & Gene Therapy. During this discussion, key stakeholders explored strategies for advancing gene editing platforms and addressing gaps in the current guidance—particularly the need to clarify the specific qualifications developers must meet to obtain platform designation. It was clear from the discussion that the FDA is emphasizing the need for early engagement—encouraging biopharma leaders to initiate dialogue with the agency as early as possible to facilitate a clear and efficient regulatory pathway. 

 The vision we are striving forward is to create economies of scope and scale by leveraging validated processes so we can accelerate the delivery of lifesaving genomic therapies to patients. This shift from discovery to design will fundamentally redefine how we develop genomic medicines, unlocking their full potential for patients.