• Among the specified targets, gene editing via the CRISPR system of dystrophin is being explored to permanently correct DMD mutations and thus restore the reading frame, allowing for the production of functional dystrophin. 282 • Similarly, CRISPR based strategies are also being investigated for facioscapulohumeral muscular dystrophy (FSHD) and merosin-deficient congenital muscular dystrophy type 1A (MDC1A) which are caused by the aberrant expression of the DUX4 gene in the muscle tissue 283 and mutation in the laminin alpha 2-chain (LAMA2) gene encoding laminin alpha 2 (Lama2) protein respectively. 284 In terms of sheer number of CRISPR-based therapeutics in the developmental pipeline, the leading organization is CRISPR Therapeutics contributing 17% of CRISPR-based therapeutics in preclinical and clinical development. With a focus on the development of transformative medicines using its proprietary CRISPR/Cas9 gene-editing platform, CRISPR Therapeutics in collaboration with Vertex Pharmaceuticals launched the first-ever US FDA approved CRISPR-based therapy Casgevy. Other key players that are actively involved in developing CRISPR-based therapeutics include Intellia Therapeutics (10%), followed by Arbor Biotechnologies (8%), and Chengdu Gene Vector Biotechnology (6%) among others (Figure ). Geographical distribution of companies engaged in CRISPR-based research and development indicates that the United States is the leader accounting for 46%, followed by China (14%) and Switzerland (12%) (Figure ). American universities, research institutions, and biotech companies have spearheaded much of the work on CRISPR technology. Biotech companies such as CRISPR Therapeutics, Vertex Pharmaceuticals and Intellia Therapeutics are major players. China has been a pioneer (14%) in applying CRISPR technology in clinical settings. The country has launched a variety of clinical trials, particularly focusing on cancer treatment using CRISPR-edited immune cells. 286