Clustered regularly interspaced short palindromic repeats (CRISPR)-based therapeutics are rapidly gaining traction for the management of infectious diseases. Programs targeting pathogens such as the human immunodeficiency virus (HIV), herpes simplex virus (HSV), and drug-resistant Escherichia coli (E. coli) are advancing in the clinic, signaling a transformative shift in the infectious disease space.
CRISPR-based therapeutics show potential to combat antimicrobial resistance (AMR) and deliver functional cures for chronic viral infections, says GlobalData, a leading data and analytics company.
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GlobalData’s latest report, “CRISPR Gene Editing in Infectious Diseases: Market Overview”, reveals that programmable gene editing technologies are now being investigated in clinical trials to address long-standing unmet needs in both viral and bacterial infections, with companies such as Excision BioTherapeutics, BDGene Therapeutics, Locus Biosciences, and SNIPR Biome leading the charge.
CRISPR-based therapies
Abigail Harris, Infectious Disease Analyst at GlobalData, comments: “CRISPR-based therapies are particularly promising for targeting latent viral reservoirs that evade traditional antivirals. Excision’s EBT-101, which removes integrated HIV DNA from infected cells, is the first CRISPR-based therapeutic of its kind to enter human trials for HIV and represents a major milestone in gene editing.”
The report also highlights how CRISPR-enhanced phage therapy is gaining traction as a means to combat AMR. Companies such as Locus Biosciences and SNIPR Biome are developing engineered bacteriophages that selectively eliminate antibiotic-resistant E. coli, with Phase I/II trials already underway.
Harris continues: “While early trial results are encouraging, key opinion leaders (KOLs) interviewed by GlobalData highlight delivery challenges, immune responses, and regulatory uncertainty as major hurdles to widespread adoption. New delivery platforms, such as lipid nanoparticles and adeno-associated viruses (AAVs), are under active investigation to enable safe, targeted in vivo editing.”
Regulatory landscape
The regulatory landscape is evolving to accommodate these innovative technologies. Agencies such as the FDA and EMA are adapting traditional frameworks to manage the unique risks and potentials of CRISPR-based platforms. However, experts emphasize the need for more streamlined, platform-based approval pathways to support rapid iteration and adaptation, especially in viral diseases prone to mutation.
Harris concludes: “Despite current challenges, CRISPR-based therapeutics hold the potential to shift the treatment paradigm for some of the most persistent infections worldwide. As more data emerges from clinical trials, gene-editing-based approaches in infectious disease are expected to see increased momentum, investment, and regulatory support.”
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