Enabling Discovery to Clinic Research with the Halo Platform

A discovery to clinic solution for a research technology has a few key attributes.

1. Research and clinical quality: the solution is capable for both cost-effective research use as well as clinical grade quality. This could be the quality or performance of a reagent, instrument, or other technology.
2. Enable scalability and diversity: the solution must be able to provide both flexible scale and support a relevant diversity of applications. Additionally, given the diverse requirements of scientific research, an effective solution
3. Demonstrate true standardization Researchers face enough challenges in biology without additional reagent or technology complications, and with poor reproducibility reagents accounting for $17B in additional annual R&D expense, standardization is an essential component. When moving through the already risk-laden stages of drug discovery research, such continuous standardization is essential.

At Synthego, we are building discovery to clinic platform solutions for CRISPR genome engineering. Through these platform solutions, we hope to enable researchers to optimally leverage CRISPR to accelerate their research workflows.

We are thrilled to introduce our first step in that direction with our proprietary CRISPR tools platform, Halo.

The Halo Platform integrates synthetic chemistry, machine learning, and automation to produce a highly scalable, standardized, and dynamic range of quality synthetic RNA products for genome engineering. Integral to this platform are continuous feedback loops enabling synthetic RNA reagents to be iteratively optimized, resulting in the best possible chemically modified sgRNA for CRISPR genome engineering.

The Halo platform powers products designed to enable researchers to confidently and optimally use CRISPR to advance their research workflows, from target identification to preclinical research to the clinic. In particular, the quality, scalability, and standardization of the platform has enabled the development of CRISPR tools with unprecedented enhancements -

The pure, synthetic RNA reagents manufactured with Halo enable high-efficiency editing in all cell types, including challenging cell types such as iPS cells or primary cells.
Halo leverages Synthego’s proprietary multi-guide gene knockout technology, the most reliable gene knockout approach on the market.
Halo enables reagent turnaround times on the order of days, even for complex and large custom reagent libraries.
Halo provides unparalleled accessibility to CRISPR, with industry-leading value for our CRISPR tools.
The above traits permeate throughout the products of Halo: sgRNA kits, multi-guide gene knockout kits, arrayed CRISPR screening libraries, and GMP-grade sgRNA.

Importantly, these products link together in various ways to empower researchers to best use CRISPR to advance their entire discovery to clinic workflows. For example, target identification performed using Synthego’s arrayed multi-guide CRISPR libraries can be validated using gene knock out kits or sgRNA. Or if considering the use of CRISPR reagents in a therapeutic, Synthego’s RUO, GMP-like, and GMP-grade sgRNA enable a seamless transition for cell and gene therapy developers, reducing the ask associated with such preclinical to clinical development.

In each of the above examples, the relevance of these products depends on the particular therapeutics workflow in question, e.g. small molecule or biologic drug discovery or cell and gene therapy. While there is significant detail with respect to how CRISPR can be optimally applied to each of these workflows (talk to a Synthego CRISPR expert to learn more), at a high level, there are a number of ways genome editing can enable discovery to clinic research workflows.

CRISPR can accelerate progress across the entire process of discovery of small molecules or biologics. This impact can be seen from the early stages of target identification and validation to the development of biologically-relevant disease models for lead identification and preclinical research.

The key is to optimally use CRISPR approaches to move through the research process seamlessly, confidently, and cost-effectively.

Synthego has developed the offerings—across CRISPR reagents and cell engineering—and the expertise to partner with researchers and companies across their entire research workflows. From target identification through preclinical development, we can help you optimally apply genome engineering solutions to advance your research.

In the world of cell and gene therapy, genome editing is being leveraged to revolutionize the field. From fixing genomic mutations associated with disease to modifying cells to attack cancer, to creating edits with regenerative effects, genome editing is poised to overcome many of the challenges inherent to engineering cell and gene therapies. Primary issues amongst these challenges are the manufacturing costs of viral engineering approaches, risks associated with tumorigenesis, and enhancements through multiplex and site-specific engineering

Synthego’s high quality, synthetic CRISPR sgRNA products seamlessly move your cell and gene therapy research from discovery to the clinic. From RUO reagents to GMP-like to GMP, we help navigate the complexities of and reduce the risk associated with gene-modified cell and gene therapy development.