David Lynch Name: Kevin Whittlesey

Where do you work? 4D Molecular Therapeutics; Emeryville, CA.

How long have you been working on FA and who was the first fellow FA researcher you met? I first interacted with FARA about 4 years ago. The staff at FARA (specifically Jen Farmer and Jane Larkindale) were incredibly helpful at providing insight into the current state of research in FA and how best to help meet the needs of people living with FA.

What got you interested in FA research? There is tremendous unmet medical need in FA which presents a significant opportunity for gene therapies to help people living with FA.

What question or challenge were you setting out to address when you started this work? 4D Molecular Therapeutics is a gene therapy company using a Therapeutic Vector Evolution process to identify and select gene therapy viral vectors that are targeted to particular tissues in the body to improve outcomes for the people living with genetic conditions.

What research topics or questions are you currently focused on? 4DMT continues to be focused on using our Therapeutic Vector Evolution platform to invent evolved vectors for the delivery of genes to specific tissue types that are affected by the diseases that we are addressing. We have observed that our targeted and evolved vectors were well tolerated and achieved enhanced delivery, increased transgene expression, reduced immunogenicity, and/or improved antibody resistance when compared to conventional AAV vectors.

What do you hope to achieve or what excites you in FA research? I am excited by the unusually wide range of preclinical animal models available for FA. The FA research community has developed important resources to enable discovery and development of new therapies. This is not the case for many other conditions.

If you have met someone living with FA, please tell us about that interaction. Did it have an impact on your work? Several years ago, 4D Molecular Therapeutics employees assembled a team to participate in and raise money for the rideATAXIA fundraiser bike ride in northern California. We had the opportunity to meet a number of people living with FA, including FARA Ambassador Kyle Bryant. That was the first opportunity for many of us to meet people living with FA. It was incredibly inspiring to hear their stories and to hear their tremendous appreciation for the ongoing scientific research.

You serve voluntarily on FARA’s Scientific Advisory Board. Please tell us what you see as FARA’s key role in the research process. FARA serves a very important role in the research process. FARA is uniquely positioned to understand what aspects of the disease are most important to those living with FA and where the important unmet needs are, and then to interface with the research community to ensure that the research is aligned with addressing those need.

Tell us more about yourself and/ or your journey with FA research. My particular role and expertise is in the preclinical stage product development. I find it incredibly rewarding to work at that interface, striving to efficiently move new products from preclinical testing to clinical trials.