8SEPTEMBER - 2021TECH OUTLOOKGene therapies have long held immense promise for the correction of genetic mutations that induce disease states, as well as for the expression of proteins or RNAs that hold therapeutic value inside diseased cells. The most recently discovered tools for genome editing, more specifically the CRISPR technologies, have come to the forefront of clinical efforts at developing newer, better therapeutics. Between 2014 and 2018, NIH funding for CRISPR related research has increased by almost 100 times, to over $1 billion[1], and, according to information available on PubMed, the percentage of CRISPR-focused publications focused on therapeutic applications has catapulted from around 3 percent in 2015 to over 16 percent in 2019. In addition, a review of clinical trial data available on clinicaltrials.gov show that the first clinical trials for CRISPR have demonstrated tangible patient benefits, with four CRISPR programs in Phase II trials in the U.S. alone. As academic research continuously advances towardclinical development and to the patients, both the challenge and potential for advancement of manufacturing processes related to these new therapies are growing. CRISPR-based therapeutics can be delivered to patients in a number of ways. Some, using viral vectors, have well developed manufacturing processes that undergo constant optimization by companies like MilliporeSigma. Other therapies, specifically the direct delivery of ribonucleoprotein complexes into either cells for ex vivo therapies, or patients for in vivo therapies, will require substantial optimization to economically deliver to patients at scale.Challenge 1: Integrated manufacturing of gene therapy APIsTo date, most non-viral gene therapy components are manufactured piecewise (RNA, protein, delivery vehicle, etc.) and assembled by the facility administering the therapy. Scaling this sort of a quasi-manufacturing process to cost-effectively reach consumers is nearly impossible. Instead, the manufacturers and contract development and manufacturing organizations (CDMOs) will have to expand their validation processes to include more diverse range of componentsand molecules than they have in the past. The companies supplying these types of modalities will have to learn to think about drug products more holistically. While majority of manufacturers of these types of materials currently focus on only a fraction of the necessary drug substance; rapid, scalable By Benjamin Borgo, Head of Portfolio Management for Genome Engineering and Modulation, MilliporeSigmaIN MY OPINIONCRISPR Technology: Exploring the Promise & Challenges of Genome Editing
< Page 7 | Page 9 >