I recently accepted the challenge to join the Timmerman Traverse for Sickle Forward campaign and others in the biotech community to climb Mount Kilimanjaro (Elevation 19,340 ft) in September 2024 to raise awareness for sickle cell disease (SCD). SCD is an inherited blood disorder that affects over 6 million people worldwide. In persons affected with this disease, red blood cells become “sickled” shaped and can block blood flow to deprive tissues of oxygen. These vaso-occlusive crises can result in extreme pain, so extreme that hospitalization is often required just to manage the pain. This is a debilitating disorder that can significantly lower life expectancy and quality years of life.

For the past 10 years, I have dedicated my career to the discovery and development of medicines for genetic diseases such as SCD. My journey has included stops at Intellia Therapeutics to advance CRISPR-based genetic medicines and Liberate Bio, a company I co-founded to develop vehicles that enable precise delivery of genetic medicines to various tissues and organs. SCD is a high priority indication for Liberate. Imagine a world where one day we will be able to administer a curative, genetic medicine directly into a SCD patient and that medicine will traffic to blood stem cells to correct the sickle cell mutation – all from a single shot into the arm! These types of pursuits are what get me out of bed in the morning!

Lacking access to adequate health care, it is estimated that 50-80% of children born with SCD in Africa will die before the age of 5. Our team will aim to collectively raise $1M to support Sickle Forward’s initiative to increase diagnosis and basic treatment for newborns throughout Africa to provide better outcomes. In addition, a gracious donor has agreed to match $1M in funds for SCD research and treatment in the US.

I have set a personal goal to fundraise $50,000. Will you join me on this journey?