Few fields in medicine are as promising and complex as gene therapy. As researchers race to develop cures for previously untreatable genetic conditions, Srinivasa Reddy Mukkala, with his multidisciplinary background, is crucial in translating scientific breakthroughs into approved therapies, potentially changing the lives of countless patients worldwide.
Educational Background and Career Progression
With a solid foundation in pharmacy from Dr. M.G.R. Medical University in Chennai, Mukkala expanded his knowledge base with a master’s in chemistry from Lamar University, Texas, and later pursued a master’s in information systems. This combination of pharmacy, chemistry, and information technology has made Srinivasa Reddy Mukkala a versatile professional.
Starting as a Statistical Programmer, Srinivasa Reddy Mukkala quickly rose through the ranks, holding positions such as team lead at MMS Holdings and Associate Director at Bluebird Bio. His progression culminated in his current role as director of statistical programming and biostatistics at Episdata Incorporated, where he leads the life sciences division.
“My background has been instrumental in understanding the nuances of drug development from multiple perspectives,” Srinivasa Reddy Mukkala shares. “It has allowed me to bridge scientific research and data analysis, which is a non-negotiable in our industry.”
Leading Food and Drug Administration Approvals
“Each FDA approval brings us closer to improving patient lives. It is about data analysis and making sure potentially life-changing therapies reach those who need them most,” Srinivasa Reddy Mukkala says.
He has led several significant Food and Drug Administration (FDA) approvals, showcasing his skills in dealing with the regulatory landscape. One of his notable achievements was leading the statistical programming for Omadacycline (NUZYRA), a tetracycline-class antibacterial approved by the FDA in 2018 for treating bacterial pneumonia and skin infections.
Building on this success, Srinivasa Reddy Mukkala played a role in the approval of Zynteglo, the first FDA-approved gene therapy for Beta-Thalassemia. As the lead for submission activities, he contributed significantly to its FDA approval in August 2022. His involvement extended to supporting FDA Advisory Committee meetings, providing insights into submission standards and addressing regulatory questions. This self-inactivating lentiviral vector-based therapy represented a new way of treating genetic disorder, Transfusion-dependent beta thalassemia (TDT) which requires lifelong blood transfusions to treat severe anemia. This one time treatment has been a lifesaving therapy for many patients to achieve transfusion independence and improve their quality of life.
Srinivasa Reddy Mukkala also contributed to another life threatening new gene therapy submission: LYFGENIA for Sickle Cell Disease. This therapy, approved in December 2023, established his name in gene therapy submissions. It is a one time gene therapy treatment specifically designed for sickle cell disease, which works by modifying a patient’s own blood stem cells to produce healthy, non-sickling hemoglobin, potentially reducing or eliminating vaso-occlusive pain crises associated with the disease.
Statistical Analysis System (SAS) Expertise in Rare Disease Research
Srinivasa Reddy Mukkala’s proficiency in SAS programming has been key to his contributions to rare disease research. As an SAS Certified professional, he has used this technical skill to analyze clinical trial data, particularly in gene therapy and rare diseases. “In rare disease research, every data point counts,” says Srinivasa Reddy Mukkala.
In his current role at Episdata Inc., Srinivasa Reddy Mukkala continues to apply his SAS skills across multiple therapeutic areas. His work involves developing data standards, analysis, and reporting specifications while adhering to FDA and Clinical Data Interchange Standards Consortium (CDISC) guidelines. This meticulous method guarantees that the data submitted for regulatory review is of the highest quality, potentially speeding up the approval process for new therapies.
“Our SAS analyses help in understanding the efficacy of potential treatments and in identifying patterns that might lead to advances in these understudied conditions.”
A Bright Future for Gene Therapy
Srinivasa Reddy Mukkala also helps improve FDA submission standards as a member of the Contributions to Clinical Data Interchange Standards Consortium (CDISC.org) wiki and analysis data standards development team.
Recognition for Srinivasa Reddy Mukkala’s contributions has come in various forms. He has received awards for his role in successful submissions, including accolades for his work on Zynteglo at Bluebird Bio and Omadacycline at MMS Holdings—honors that reflect his technical skills, leadership, and ability to guide teams through submission processes.
“Being part of standard development is about preparing our industry for the future,” Srinivasa Reddy Mukkala says. “It is gratifying to know that the standards we develop today will facilitate the approval of life-saving therapies tomorrow.”
His work will undoubtedly influence the future of gene therapy. “The future of medicine lies in personalized, genetic-level treatments,” Srinivasa Reddy Mukkala caps off. “My goal is to help new therapies reach patients as quickly and safely as possible.”
Photo courtesy of Srinivasa Reddy Mukkala
