Verve Therapeutics

Eli Lilly will acquire gene-editing startup Verve Therapeutics for up to $1.3 billion, including an upfront payment of almost $1 billion and $300 million in milestone-based payments.

Novartis's LEQVIO (inclisiran), the first FDA-approved siRNA therapy for LDL-C reduction, is projected to reach USD 2.2 billion in US market size by 2034, offering a novel mechanism with biannual dosing that enhances patient adherence.

Eli Lilly has made a $1.3 billion investment commitment to Verve Therapeutics, marking a significant bet on cardiovascular gene therapy development.

Peter Marks, head of FDA's Center for Biologics Evaluation and Research since 2016, has resigned, leaving cell and gene therapy developers without their biggest regulatory advocate during a challenging investment period.

CRISPR Medicine News tracks approximately 250 clinical trials involving gene-editing therapeutic candidates as of February 2025, with more than 150 trials currently active across diverse therapeutic areas.

• Verve Therapeutics received FDA clearance to expand its VERVE-102 trial into the US, potentially offering a one-time base editing treatment for persistent high cholesterol conditions. • The company's gene editing approach targets the PCSK9 gene to deliver lifelong cholesterol lowering, distinguishing it from current therapies that require regular injections ranging from bi-weekly to semi-annually. • Initial safety and efficacy data from the Phase 1b Heart-2 trial is expected by the end of June, with dose escalation data and Phase 2 trial initiation planned for later this year.

• Pharmaceutical companies warn in SEC filings that Trump administration's FDA staffing and budget cuts could significantly delay or halt new drug approvals and commercialization processes. • Recent layoffs of hundreds of FDA employees have sparked industry-wide concerns about disruptions to clinical trials, grant applications, and regulatory oversight activities. • Multiple biotech firms, including Xenon Pharmaceuticals and Rezolute, report that reduced FDA capacity could negatively impact their drug development timelines and business operations.

Verve Therapeutics anticipates initial data from the Heart-2 Phase 1b trial of VERVE-102, targeting PCSK9, in Q2 2025, including safety and efficacy data.

• Beam Therapeutics reported a patient death in their BEAM-101 sickle cell disease trial, likely due to the conditioning regimen. • AstraZeneca scientists engineered PsCas9 for therapeutic genome editing in mouse liver, showing promise for hypercholesterolemia treatment. • YolTech Therapeutics' novel LNP system delivers base editor mRNA to bone marrow cells, activating foetal haemoglobin production for blood disorder treatment.

Rocket Pharmaceuticals completed enrollment in a Phase 2 trial of RP-A501 gene therapy for Danon disease, targeting LAMP2 expression and left ventricular mass reduction.