Beam Therapeutics

Recent studies presented at the ASH Annual Meeting highlight advancements in treating sickle cell disease (SCD) and immune thrombocytopenia (ITP). Key findings include a new oral medication for ITP showing durable platelet response, etavopivat reducing SCD pain episodes by nearly half, hydroxyurea benefits for HbSC SCD variant, safe fertility preservation for SCD patients, and promising early results from a base-edited gene therapy for SCD.

Korro Bio received approval for a Phase 1/2a trial of KRRO-110, an RNA-editing therapy for alpha-1 antitrypsin deficiency (AATD), targeting the PiZ mutation in SERPINA1. KRRO-110 aims to correct the mutation using an RNA-editing enzyme, ADAR, encapsulated in a lipid nanoparticle, with preclinical data showing sustained editing efficiency and increased AAT protein levels. The REWRITE trial will evaluate safety and tolerability in healthy adults and AATD patients, with dosing expected in Q1 2025.

The biopharma sector has seen 955 deals worth $114.5 billion through early August 2022, with deal values up but numbers down compared to previous years. Immunotherapies and RNA technologies drive major deals, including a $6.2 billion agreement between Poseida Therapeutics and Roche. Despite a volatile economy, the industry focuses on transformative therapies, with significant investments in curative treatments and a shift towards venture capital financing. FDA approvals are down, but the sector anticipates future breakthroughs in patient care.

FDA investigates bluebird bio's Skysona gene therapy after reports of secondary blood cancers in treated patients, linked to clonal vector insertions and gene mutations. The FDA advises considering alternative therapies and is evaluating further regulatory action.

The global CAR T-cell therapy market is projected to grow from $2.7B in 2023 to $10.8B by 2030, driven by tech advancements, expanding clinical applications, and rising cancer prevalence. Key trends include the move into solid tumors and development of allogeneic therapies, though challenges like side effects and high costs persist.

The global CAR T-cell therapy market, valued at US$2.7 billion in 2023, is projected to grow to US$10.8 billion by 2030, driven by technological advancements, expanding clinical applications, and the demand for personalized cancer treatments. Key growth areas include treatment for solid tumors and allogeneic therapies, despite challenges like high costs and side effects.

Cathie Wood sees transformative potential in biologics, driven by AI, genetic sequencing, and gene editing, exemplified by breakthroughs in sickle cell disease treatment and AI-accelerated drug development, despite market volatility.

CRISPR Therapeutics stock is down 47% since March due to slow Casgevy launch, but it has five other therapy candidates in clinical-stage testing. Despite slow sales, Casgevy's potential and a $1.9 billion cash reserve make CRISPR Therapeutics a risky but potentially lucrative investment.