Verve Therapeutics

CRISPR Gene Editing Breakthrough Saves Baby with Ultra-Rare Metabolic Disorder

9 days ago

• Doctors at Children's Hospital of Philadelphia successfully treated a baby with severe CPS1 deficiency using a personalized CRISPR base-editing therapy, marking a first-of-its-kind approach for this rare metabolic disorder. • The experimental treatment, developed within just six months of diagnosis, corrected the infant's specific genetic mutation by delivering edited DNA to liver cells via lipid nanoparticles, allowing him to reduce medication and process more dietary protein. • This breakthrough demonstrates the potential for creating customized gene therapies for millions with rare genetic diseases, with researchers suggesting costs comparable to liver transplantation and possibilities for treating numerous other conditions.

LEQVIO Emerges as Leading PCSK9 Inhibitor in Cholesterol Management Across Major Markets

16 days ago

• 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. • The PCSK9 inhibitor market, valued at USD 2 billion across seven major markets in 2023, is expected to grow substantially due to expanding applications in statin-intolerant patients and preventive cardiovascular strategies. • Emerging competitors including Lerodalcibep, Merck's oral MK-0616, and Verve Therapeutics' gene-editing candidates are advancing through clinical trials, potentially disrupting LEQVIO's market position after 2027.

Vertex Pharmaceuticals Abandons AAV Vector Technology Amid Broader Industry Retreat from Gene Therapy

17 days ago

• Vertex Pharmaceuticals has discontinued all research on adeno-associated virus (AAV) vector technology, impacting partnerships with Affinia Therapeutics and Tevard Biosciences focused on Duchenne muscular dystrophy treatments. • The retreat from AAV vectors follows a broader industry trend, with Pfizer, Roche, Takeda, and Biogen all scaling back gene therapy programs due to safety concerns, limited payload capacity, and high manufacturing costs. • Despite industry pullback, companies like Affinia Therapeutics continue developing next-generation AAV vectors, while others explore alternative delivery systems such as Herpes simplex virus-1 vectors with larger genetic payloads.

FDA's Cell and Gene Therapy Champion Peter Marks Departs, Leaving Industry at Critical Juncture

2 months ago

• 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. • Under Marks' leadership, the FDA approved dozens of cell and gene therapies including the first gene therapy, first cellular treatment for cancer, and first CRISPR gene editing medicine, establishing flexible regulatory frameworks for these novel treatments. • Despite concerns about regulatory uncertainty following Marks' departure, incoming FDA Commissioner Marty Makary has signaled support for conditional approval pathways for rare disease treatments where randomized controlled trials aren't feasible.

Verve Therapeutics Expands Base Editing Trial for Cholesterol Treatment into US Following FDA Clearance

2 months ago

• 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.

Beam Therapeutics Achieves Breakthrough in Base Editing for Alpha-1 Antitrypsin Deficiency

3 months ago

• Beam Therapeutics' BEAM-302 demonstrated successful DNA correction in alpha-1 antitrypsin deficiency patients, marking the first clinical proof of concept for direct mutation correction using base editing technology. • The treatment increased properly folded AAT protein levels up to 2.8 times baseline with a 78% reduction in misfolded protein in one high-dose patient, potentially addressing both liver and lung manifestations of the disease. • Initial safety data from the nine-patient trial appears favorable, with the company now planning to test higher doses and expand enrollment to include patients with mild-to-moderate liver disease.

FDA Budget Cuts Under Trump Administration Threaten Drug Development Pipeline

3 months ago

• 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.

Novel Gene-Silencing Therapy Shows Promise for Long-Term Cholesterol Control with Single Dose

3 months ago

• Groundbreaking epigenetic editing technique demonstrates over 98% reduction in PCSK9 levels and 70% decrease in LDL cholesterol through a single treatment in preclinical studies. • The novel PCSK9-epigenetic editor (PCSK9-EE) therapy maintains its effectiveness for over a year without permanently altering DNA, offering a potential alternative to daily cholesterol medications. • Research in non-human primates shows promising safety profile with only transient liver enzyme elevations, while maintaining the ability to reverse the treatment if needed.

Verve Therapeutics Advances Pipeline with Focus on Gene Editing for Cardiovascular Disease

4 months ago

• 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. • VERVE-301 has been nominated as the development candidate targeting the LPA gene, triggering a milestone payment from Eli Lilly, who will fund Phase 1 development. • The Pulse-1 Phase 1b trial for VERVE-201, which targets ANGPTL3, is progressing, with an update expected in the second half of 2025. • Verve's strong financial position, boosted by the Lilly milestone payment, extends the company's cash runway into mid-2027.

Axsome's AXS-05 Shows Promise in Alzheimer's Agitation Trials, NDA Submission Planned for 2025

5 months ago

• Axsome Therapeutics' AXS-05 demonstrated a statistically significant delay in agitation relapse in Alzheimer's patients in the ACCORD-2 Phase 3 trial. • The ADVANCE-2 trial did not meet its primary endpoint, but results numerically favored AXS-05, with a good safety profile across trials. • Axsome plans to submit an NDA to the FDA in the second half of 2025, supported by data from four Phase 3 trials. • AXS-05 has Breakthrough Therapy designation, potentially expediting its review for Alzheimer's disease agitation, a condition with limited options.

Cytokinetics' Aficamten Advances in Regulatory Review for Obstructive Hypertrophic Cardiomyopathy

6 months ago

• The EMA has validated Cytokinetics' Marketing Authorization Application (MAA) for aficamten, a cardiac myosin inhibitor, for treating obstructive hypertrophic cardiomyopathy (HCM). • The FDA has accepted the New Drug Application (NDA) for aficamten with a PDUFA target action date of September 26, 2025, and no advisory committee meeting is planned. • Aficamten significantly improved exercise capacity and clinical outcomes in the SEQUOIA-HCM Phase 3 trial, supporting regulatory submissions in the U.S., Europe, and China.