Going from Bench to Bedside: Partnerships for Gene Therapy Innovation

Igi Beacon Partnership

From homemade chicken noodle soup to zinc and vitamin C supplements, immune boosters for fighting off disease become popular every cold and flu season. While human adaptive immunity is a complex immunological system, even organisms as small and simple as bacteria hold acquired immunity to viral attackers. Originally discovered as an enigmatic set of repeated chromosomal DNA sequences in E. coli¹, CRISPR-Cas systems have been one of the most revolutionary biological discoveries of the last century. Transcribed products of these sequences and associated systems can be “reprogrammed” to edit DNA².

The discovery that CRISPR-Cas complexes can be selectively reprogrammed to target, cut, and repair dsDNA to create custom genome editing has changed what is possible in life sciences research and development and has brought custom gene therapies from the frontiers of biology to pharmaceutical pipelines. Despite intensive investment and research, many gene therapies have yet to reach patients. Challenges and bottlenecks remain in research, process development, delivery and manufacturing of gene therapies. Partnership and collaboration across academics, industries, and government institutions are needed to help gene therapies realize their promise.

Manufacturing CRISPR-based gene therapies remains a major obstacle to bringing gene therapies to patients. The potential is enormous, but ensuring safety, manufacturing and scaling of these treatments remains a bottleneck. Like small molecule therapies and antibody-based therapeutics in the past, a way to industrialize manufacturing of CRISPR-based therapies and make them reproducible and profitable is required. A platform for rapid iteration and development of these therapies is needed to bring these therapies to fruition.

In January 2024, Danaher Corporation announced a historic partnership with the Innovative Genomes Institute (IGI) around creating CRISPR-based therapies for rare diseases. This partnership aims to develop CRISPR-based gene therapies to potentially cure rare diseases by developing a scalable technology for iterative development of gene therapies by leveraging the technology and expertise across both Danaher Corporation and IGI.

This partnership is not a one-off event: Jose-Carlos Gutierrez-Ramos, Senior VP and CTO of Danaher Corporation commented, “We aim to develop high-value solutions for the most critical and complex problems in biomedicine. In the service of that goal, we partner with world-leading scientists and physicians – working hand-in-hand.” To partner with Danaher Corporation and solve life’s most challenging problems, contact an expert today.

References

  1. Ishinoi, et al. 1987. Nucleotide sequence of the iap gene, responsible for alkaline phosphatase isozyme conversion in Escherichia coli, and identification of the gene product. J. Bacteriol. 169(12): 10.1128%2Fjb.169.12.5429-5433.1987.
  2. Jinek, et al. 2012. A programmable dual RNA-guided DNA endonuclease in adaptive bacterial immunity. Science. 337(6096): 10.1126%2Fscience.1225829.