Objective:
To summarize recent advancements in gene therapy and related technologies.
Approach:
- CRISPR/Cas9 and ASOs: A study by the University of Basel and Roche identified that intracellular transport speed of ASOs affects their therapeutic impact, with CRISPR/Cas9 screening pinpointing genes like AP1M1 that influence ASO activity.
- AbbVie Acquisition: AbbVie announced the acquisition of Capstan Therapeutics for up to $2.1 billion to enhance its pipeline in immunology and cell therapy.
- CRISPR Therapeutics Recognition: CRISPR Therapeutics was named to TIME’s 2025 list of the 100 Most Influential Companies for its work in gene editing and expanding its focus to more prevalent conditions.
- Shift Bioscience Innovations: Shift Bioscience introduced a refined ranking system for virtual cell models to improve gene target discovery in aging research.
- Normalization of SCD Hemodynamics: A study assessed the impact of CRISPR-Cas9-based gene therapy on cerebral blood flow in adults with sickle cell disease, showing significant reductions in CBF and no new neurological complications.
Key Findings:
- Inhibiting AP1M1 slows endosomal transport of ASOs, increasing their effectiveness.
- AbbVie aims to diversify its pipeline through the acquisition of Capstan Therapeutics.
- CRISPR Therapeutics is expanding its focus to make gene editing treatments more accessible.
- Shift Bioscience's new metrics aim to enhance gene target discovery in aging research.
- Gene therapy in SCD showed durable improvements in cerebral hemodynamics.
Interpretation:
Limitations:
- The study on SCD involved a small cohort, limiting the generalizability of the findings.
Conclusion:
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