Suma Krishnan's Revolutionary Approach to Gene Therapy Delivery
With an extensive background spanning decades in pharmaceutical research and drug development, Suma Krishnan has firmly established herself as one of the most influential and pioneering innovators in contemporary biotechnology. In her late forties, she began to critically question a fundamental assumption that had long dominated the field of gene therapy. Why must treatment always be administered through injection into the body? Krishnan held a strong conviction that genetic medicine could potentially be applied directly to the skin, a novel idea that would ultimately lead to a groundbreaking, first-of-its-kind therapy for a devastating and rare genetic disorder.
Founding Krystal Biotech and Achieving a Medical Milestone
In 2016, at the age of 51, Suma Krishnan co-founded Krystal Biotech alongside her husband and long-time scientific collaborator, Krish Krishnan. Several years later, the company achieved a significant medical milestone that earned Suma Krishnan a prestigious place on the Forbes 250 America's Greatest Innovators list. This honor specifically recognizes ideas and individuals that possess the power to reshape entire industries through transformative innovation.
Focus on Epidermolysis Bullosa: The Butterfly Skin Disease
Krishnan's pioneering work is centered on Epidermolysis bullosa, commonly referred to as butterfly skin disease. This is a rare, inherited condition characterized by extremely fragile skin. Even minimal friction or minor trauma can cause painful blisters and open wounds. A significant number of patients are children who endure constant pain and are susceptible to frequent, serious infections.
For many decades, treatment options were severely limited and primarily palliative. Standard care focused on:
- Meticulously bandaging wounds
- Managing chronic pain
- Preventing and treating infections
Critically, there was no available therapy that addressed the underlying genetic cause of the disease itself.
A Paradigm Shift in Delivery: Topical Gene Therapy
Suma Krishnan's key insight was not about discovering gene therapy as a concept, but rather about revolutionizing how it could be delivered. She proposed and developed a topical gene therapy that could be applied directly to open wounds. This innovative approach would deliver a healthy, functional copy of the faulty gene directly to the skin cells where it was most urgently needed.
At the time, this concept faced considerable skepticism from many experts in the field. Gene therapies were widely believed to be difficult to administer safely on a repeated basis. Krishnan, however, firmly disagreed with this prevailing view. "The challenge was not just delivering the gene once," she has explained in interviews. "It was making sure patients could use it safely again and again over time."
From Concept to FDA Approval: The Vyjuvek Story
This visionary idea materialized into Vyjuvek, a topical gene therapy specifically designed for dystrophic epidermolysis bullosa. In a landmark decision in 2023, the US Food and Drug Administration granted approval to Vyjuvek. This approval marked several historic firsts:
- It became the first-ever FDA-approved topical gene therapy.
- It was among the first gene therapies cleared for any skin disease.
Clinical trials demonstrated meaningful and significant wound healing with repeated use of the therapy, directly addressing long-standing concerns regarding both safety and treatment durability. For patients, their families, and healthcare providers, this approval represented a monumental shift from merely controlling symptoms to offering a targeted, genetic treatment.
Recognition and the Path to a Multi-Billion Dollar Company
Forbes recognized Suma Krishnan for her exceptional achievement in transforming a high-risk scientific hypothesis into a fully approved medicine. Under her leadership, Krystal Biotech has grown into a multi-billion-dollar biotechnology company. The gene therapy platform developed for Vyjuvek is now being actively explored for potential application in treating other skin conditions and genetic disorders, showcasing the platform's broader utility.
Industry analysts frequently highlight Krishnan's deep and comprehensive experience in drug development, which she accumulated for decades prior to founding her company. This extensive background proved invaluable in navigating the long, complex timelines and stringent regulatory pathways inherent to bringing a novel therapy to market.
Challenging Assumptions About Age and Innovation
Suma Krishnan's story also powerfully challenges conventional assumptions about age and the peak of innovative potential. She founded Krystal Biotech in her fifties and, years later, successfully delivered an FDA-approved therapy. Many editors at Forbes have noted that some of the most impactful and world-changing innovations often emerge after long, distinguished careers, rather than at their very beginning.
A Lasting Impact on Rare Disease Care and Scientific Thought
Today, Suma Krishnan's work stands as compelling proof that persistence, scientific rigor, and challenging established norms can yield transformative results. For the countless families affected by epidermolysis bullosa, the impact of her work is profoundly personal and life-altering. For the broader biotechnology and scientific community, it opens entirely new avenues for thinking about gene therapy delivery mechanisms.
Krishnan has consistently stated that the goal was never personal recognition or accolades. "What truly matters," she has emphasized, "is whether patients' lives actually change for the better." For many individuals living with the daily challenges of butterfly skin disease, that positive change has already begun, thanks to her visionary work and unwavering dedication.
