How did you first become involved in ophthalmology research?

My PhD research focused on using pluripotent-based models to understand oligodendrocyte biology in health and disease. I leveraged these in vitro tools to identify therapeutic candidates for myelin disorders through high-throughput compound screening and optimizing CRISPR/Cas9 and oligonucleotide-based drugs. These efforts led to several awards, patents, and preclinical drug candidates that are in clinical trials (NCT06150716) or expected to advance into clinical trials in the near term. My goal is to leverage this expertise to create a rapid diagnostic and preclinical drug pipeline for orphan inherited retinal diseases. Consistent with that aim, I became involved in projects to support my scientific transition to the retinal disease space.

What is the focus of your current research?

I am currently working on three projects. In the first, I am identifying novel mutations and describing clinical phenotypes in patients with undiagnosed inherited retinal diseases. I also plan to collect tissues from these patients to build a human patient induced pluripotent stem cell (iPSC) library for future in vitro characterization and therapeutic development. Separately, over the coming year I plan to generate iPSC-derived retinal organoids from human patients with CTX-related inherited retinal disease and use these tissues to establish a preclinical data package for a novel adeno-associated virus–based gene therapy for this disease. Finally, I am leading a multicenter, retrospective trial examining the role of GLP-1 receptor agonists in the treatment of idiopathic intracranial hypertension.

Who is/are your research mentor/mentors?

Raj Apte, MD, PhD; Shiming Chen, PhD; and Margaret Reynolds, MD, at Washington University. I also keep in close contact with my PhD mentor, Paul Tesar, PhD, at Case Western Reserve University, who will almost certainly be a lifelong advisor.

How do you typically find a research opportunity or identify a project you would like to pursue?

I try to seek guidance from experienced mentors who may have invaluable insights into emerging research ideas and potential projects. I also keep abreast of the literature to help me identify areas that warrant further investigation. From there, I try to be strategic and filter out projects where I don’t personally have an interest in the question, the impact of the work would be too low for the time investment, or where various constraints make the project infeasible. To the latter point, research funding is typically a barrier to address many exciting questions, so identifying grant opportunities or fellowship awards is often critical in this process. Sometimes it feels like the stars must align to actually pull off something truly groundbreaking, but when you are responsible for a significant advancement in a field, it is incredibly rewarding.

What do you look for in a research partner, either on the physician or industry side?

On the academic side, finding a collaborator who is enthusiastic, capable, and supportive is key. You want someone who shares your vision and is willing to contribute a complementary skill set to improve the overall project. Look at their prior publication history to see if they can fill in any knowledge or expertise gaps. Ask prior collaborators about their experiences with the individual. Try to set expectations early in terms of roles, timelines, and authorship order, and, if you see any red flags, address them or pivot to another partner as early as possible. There is a lot of teamwork involved in research projects, and you want to make sure the process is as constructive as possible.

On the industry side, identifying companies that are truly passionate about helping patients is paramount. This should be evident by a track record of patient-centered product development and a commitment to genuine collaboration with researchers. Put simply, when the CEO talks about patients at a far greater frequency than they do profits, it’s a probably a sign they’ll be a good partner!

What areas of research excite you most about the future of ophthalmology?

Regenerative medicine approaches using pluripotent-derived cells. These tissues have the potential to provide disease-agnostic treatments for many ocular diseases. In particular, in the retinal disorder space, we’re beginning to see some exciting trials and data that suggest progress on the technical hurdles that have previously held back these technologies. If these issues are solved, it will usher in an exciting era for the management of retinal disease, where previously incurable pathology may finally be treatable.

What are your overall research goals?

As mentioned earlier, my long-term goal is to create a diagnostic and therapeutic development pipeline for undiagnosed or orphan inherited retinal diseases. On the therapeutic side, there are several next-generation technologies that are truly modular (ie, gene therapy, CRISPR, RNA interference and antisense oligonucleotides, and cell-based therapies); once fully proven in the clinic, these technologies can be relatively easily applied in most (and in some cases all) inherited retinal diseases where the causative mutation is known. Given the potential for a straightforward pathway of treating these patients, I want to make sure we’re getting as many of these technologies to patients as possible, especially those with unique pathologic variants who might otherwise be overlooked.

What advice would you give to other aspiring or young ophthalmologists looking to conduct research?

Get involved with a question or topic that excites you. The research process can be emotionally taxing, so being passionate about your work will keep you motivated during the more difficult times. Also, don’t be dissuaded if you don’t come across the perfect project on day one. Give yourself time to become familiar with a topic and the unanswered questions in that space. As you build your knowledge, you will inevitably come up with ideas for projects. If you want to speed up the process, try to lean on your mentors to help brainstorm research questions; they usually have many exciting ideas and can provide a good starting point.