The Gene-Editing Revolution with Verve Therapeutics

Описание: Please note: as of 12/31/21, ARK’s clients own greater than 1% of the shares outstanding of Verve Therapeutics.

On this episode of FYI, ARK Analyst Ali Urman is joined by Verve Therapeutics CEO Sek Kathiresan and Chief Scientific Officer Andrew Bellinger. Verve, a biotechnology company, was created with the sole focus of protecting the world from heart disease. For many years, institutions have approached cardiovascular disease with a chronic care model, prescribing medications to help reduce symptoms and complications, such as heart attack and high blood pressure. Verve Therapeutics wants to change that model. Founded in 2018, Verve was created with the idea that we can develop a one-and-done gene-editing medicine to permanently lower LDL cholesterol to treat heart attack, the world’s leading cause of death.

In today’s episode, Dr. Kathiresan and Dr. Bellinger weigh in on the development of gene-editing and its impacts on cardiovascular disease. They discuss gene-editing costs, the importance of lowering LDL cholesterol levels, importance of liver delivery, their PCSK9 program, and why it could be revolutionary for the future of cardiovascular health. For the past four years, Verve has worked to develop proof of concept in monkeys specifically. Gene-editing therapies, such as Verves, could help create longer term health for patients. Listen in to learn more!

Key Points From This Episode:

• An introduction to Verve Therapeutics
• Dr. Kathiresan’s inspiration for treating cardiovascular disease
• Issues with the chronic care model
• Development of PCSK9
• Overcoming the unmet need of the LDL care
• Thinking about healthcare from the upstream approach
• How COVID vaccines have shown feasibility in development
• How the pipeline continues emerging
• Challenges of nanoparticles being picked up by the liver
• Developing a new therapy with homozygous monkey model
• Difference between healthy vs. heterozygous patients
• How costs affect the work and role of COVID MRNA
• Off-target editing
• What we need to get to market quicker
• Engaging with Twitter to advance the conversation
• Data visualization

Tweetables:

“When people think about gene-editing they are immediately thinking rare disease pricing and millions of dollars per dose … that’s not going to be our model because we ultimately want to reach millions of patients” – @skathire

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