CRISPR Therapeutics, Inc.

Clarivate's 2024 Drugs to Watch report highlights 13 new therapeutics expected to achieve blockbuster status by 2029, targeting conditions like sickle cell disease, RSV, and breast cancer. It also notes the growing chronic disease market in China and the impact of new technologies like CRISPR-Cas9 and AI in drug development.

Intellia Therapeutics, a CRISPR specialist, will lay off 15% of its workforce, pausing some early-stage research to focus on its advanced drug candidate, NTLA-2001, for ATTR amyloidosis. Despite setbacks, including a discontinued partnership with Novartis, Intellia continues Phase 3 trials for NTLA-2001 and explores gene editing treatments with Regeneron.

Arbor Biotechnologies, co-founded by CRISPR pioneer Feng Zhang, is advancing gene editing by developing new tools beyond Cas9 for diseases like hyperoxaluria and ALS. With a focus on liver and CNS diseases, Arbor aims for three IND filings in three years, leveraging a broad toolbox for precise gene editing. The company celebrates recent CRISPR approvals, viewing them as a precursor to more innovative treatments, including potential one-and-done solutions and preventive gene editing in the future.

The global sickle cell disease (SCD) treatment market, valued at USD 2.5 billion in 2022, is projected to reach USD 50.45 billion by 2032, growing at a CAGR of 35.1%. North America led with a 36% market share in 2022. Growth factors include market expansion in developing regions, innovative therapies, and global health initiatives. Key treatments involve blood transfusion and bone marrow transplant, with hospitals and specialty clinics as primary end-users. Advances in genetic research and personalized medicine are driving market evolution, despite challenges like economic barriers and inadequate infrastructure in developing regions.

ALS, a fatal neurodegenerative disease, has seen limited treatment options despite its discovery over a century ago. Recent scientific advancements offer hope, with new drugs like AMX0035 showing modest benefits in slowing disease progression and extending survival. However, challenges remain in understanding ALS's biology and developing effective treatments, especially for sporadic cases without known causes. The field is witnessing increased investment and research, aiming for breakthroughs that could significantly improve patients' lives.