In the United States, many people are forced to resort to platforms like GoFundMe to pay for life-saving treatments, even as the nation’s biotech innovation leads across the globe. Take Zolgensma, a gene therapy for spinal muscular atrophy (SMA), which costs nearly $2 million per patient. Despite technological breakthroughs accelerating rapidly, access to these treatments is often not covered by insurance and still remains widely inaccessible. That’s where Dr. Tejal sees an opportunity for growth.
Dr. Tejal Aslesh Tailor is an accomplished neuroscientist and molecular biologist with years of expertise in preclinical research, gene therapy, and lipid nanoparticle (LNP)-based drug delivery systems. She holds a Ph.D. in Neuroscience and nearly a decade of expertise developing innovative therapeutic strategies for rare genetic disorders such as spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD). Today, she practices as a scientist leading in vivo gene delivery studies, histology-based analyses, and molecular characterization of novel therapeutics using LNPs. Her approach of targeting rare genetic disorders with fewer injections and lower costs has the potential to unlock a new era of healthcare driven by accessibility and equal opportunity.
“Accessibility remains one of the major obstacles for families of patients with rare disorders,” Dr. Tejal explains. “To address this obstacle, my approach from the start was to develop an effective therapy that requires fewer injections thereby leading to fewer visits to the hospital. Collaboration with key opinion leaders, clinicians, health practitioners, and industry leaders will further help in advocating the awareness around patient access.”
One example of this is Cure SMA, a non-profit organization providing a platform for SMA patients, clinicians, researchers, and pharmaceutical companies to interact and learn about advancements in treatment strategies. The goal of these individuals is to bridge the gap between innovation and impact, offering opportunities for fulfilling lives for those suffering from the condition. Creating a hub of communication is a great example of ways to propel research forward in holistic ways that actually make a difference.
Dr. Tejal feels strongly that innovation on its own isn’t enough to make a true change. Developing a drug for a rare disorder is just the beginning — if the treatment isn’t accessible, what is the point? “I hope my research inspires others to take innovation a step further to develop breakthrough treatments that can benefit our patients,” she says. “If we can model our scientific ideology of problem-solving to address this challenge, we can lay the groundwork for the next generation of founders and problem-solvers.”
Pompe disease, for example, is often fought with an enzyme-replacement therapy (ERT) which requires weekly infusions. Despite ERT being recognized as a milestone in scientific advancement, a lot of barriers remain for patients to access it. Patients need weekly infusions, which cost thousands of dollars, and access depends on insurance coverage, which varies depending on the state. Coverage of treatment costs is also impacted by pre-existing health conditions. So those without insurance or in more rural, underserved areas face barriers to this treatment that prevent it from even helping them, and if the treatment can’t help patients, what’s the point of its existence? Patient-centric, and for that matter, empathy-based healthcare needs to be pushed more to the forefront of the nation’s goals.
By focusing on research, Dr. Tejal is doing more than curing disease. She’s building infrastructure for a more productive and equitable healthcare system. Her work offers a potential roadmap integrating cutting-edge science and tech with systemic change, placing compassion at the forefront. Dr. Tejal’s vision is an America where innovation includes every patient, regardless of socioeconomic status, and where the next generation of healthcare professionals prioritizes empathy as well as ambition.
