Flexible Solutions for Gene Therapy Trials: Tailoring Transformative Care

Human Gene therapy is a technique associated with modification or manipulation of gene expression for therapeutic purposes. Luxurna was the first human gene therapy drug that was developed for an inherited condition, retinal dystrophy, and approved by the Food and Drug Administration (FDA) in 2017 (Dias et al., 2017) Since then, there have been multiple studies on gene therapy to enhance modern therapeutics.

Navitas Life Sciences Supports Efficient Clinical Trials

We bring life-saving medicines to the market through well-managed clinical trials, leveraging over 30 years of experience. Right from phase I to phase IV, we support clinical trial needs from startup to closure, and beyond, with our regulatory insights and quality approach. Our services are backed by technology & analytics that are developed specifically to meet challenges.

Navitas Life Sciences has rich experience across 550+, 20+ therapeutic areas, 2 ISO 27001- compliant Data Centers and 40+ successful GCP/non-GCP audits. Our trial expertise is augmented by our proprietary OneClinical Analytics, a platform that delivers trial oversight and key data insights that aid in better decision making for efficient clinical trial management.

Gene Therapy Market

According to a report, the global gene therapy market size in 2020 was valued at USD 2.26 billion with an estimated compound annual growth rate (CAGR) from 2021 to 2028 fixed at 20.4%. The advent of better gene delivery technologies and enhanced technological expertise have created greater interest and improved scope of treatment using such therapies.

In order to understand more about gene therapy and gene therapy clinical trials, we interviewed Dr. Atul Gupta, Associate Vice President, Medical and Scientific Affairs, Navitas Life Sciences, Dr. Narendra Chirmule, CEO, Symphony Tech, and Dr.Kirk Hammond, Professor of Medicine, UC San Diego and Founder, Renova Therapeutics recently.

Tell us about your professional background and your expertise

I am a physician with over 16 years’ experience in Clinical Research, Drug Development and clinical practice. I worked as the Global Medical Lead in more than 50 studies across diverse therapeutic areas (Oncology, neurology, infectious diseases, cardiology, vaccines, biosimilars, GI, Haematology etc), across all the phases (I-IV) for both drugs, devices and combination products.

I am actively involved in multiple DSMBs and SRCs, and have been associated with successful marketing authorization approval of breakthrough therapies including PD-1 inhibitors, V2 antagonist, multiple biosimilars, vaccines and several repurposed therapies (for MDD, narcolepsy, psoriasis).

Dr. Atul Gupta,

Associate Vice President,
Medical and Scientific Affairs,
Navitas Life Sciences

How will gene therapy support the needs of patients?

There has been unprecedented progress in nucleic acid delivery, and accurate control of the human genome, which was not considered plausible few years ago. Currently, maximum work is being done on cancers and monogenic disorders, but the attention has expanded to many other conditions like Alzhiemer’s, parkinsons, and even diabetes and coronary artery disease. GTP has already been approved by FDA and EMA for various types of cancers (especially multiple myeloma, lymphoma, leukemia, rare disorders like ADA-SCID, cerebral adrenoleukodystrophy, metachromatic dystrophy, spinal, muscular atrophy, lipoprotein lipase deficiency etc.

How difficult is it to get suitable volunteers for gene therapy clinical trials?

There are certain stringent and highly specific eligibility criteria. There is also a low patient pool for extremely rare monogenic disorders (e.g. spinal muscular atrophy, lipoprotein lipase deficiency, rare lysosomal storage diseases etc.), and increased hesitancy due to serious and unexpected risks associated with gene therapy trials.

What are some of the challenges faced in a gene therapy clinical trial?

Some of the challenges are

  • Regulatory Approval: Considering the high risks associated with gene therapy trials, many regulatory authorities have discrete requirements and additional level of check by specific Gene therapy committees (e.g. Division of cellular and gene therapies in USA, Committee for advanced therapies in Europe).
  • Study Design: Requirement of complex innovative clinical trial design
  • Experience: Need for investigators with prior experience in rare disorders, GTP can be difficult
  • Patient Recruitment: Recruitment challenges
  • Follow up: Long term follow up (5-15 years depending on vector and regulatory agency requirements)
  • Storage: Ability to receive, and store Gene therapy product appropriately
  • Monitoring: Intense monitoring of subjects throughout the study
  • Safety Requirements: Unique safety risks

What are some of the risks involved in gene therapy?


  • Short-lived efficacy: Problems with integrating therapeutic DNA into the genome and the rapidly dividing nature of many cells may prevent from achieving long-term benefits.
  • Multigene disorders – Some commonly occurring disorders, such as heart disease, high blood pressure, Alzheimer's disease, arthritis, and diabetes, are affected by variations in multiple genes, which complicate gene therapy.


  • Immunogenicity reactions
  • Use of viral vectors – Viral vectors carry the risks of toxicity, inflammatory responses, and gene control and targeting issues.
  • Insertional mutagenesis
  • Off target effects
  • Neoplastic potential

Economic: High cost

How is Navitas Life Sciences equipped to conduct a gene therapy clinical trial?

We have experience in conducting complex innovative trial designs in oncology and rare diseases and have an in-depth expertise on global regulatory requirements (North America, Europe, Asia pacific and the middle east)

We have a team of highly experience professionals including medical leads, operations, data management in advanced therapies and interact with investigators with experience in complex trials.

We also have a vast pool of volunteers and have the right experience to mitigate risk due to the pandemic.

Tell us about your professional background and your expertise

I am the co-founder and CEO of SymphonyTech Biologics, a data analytics company focused on engineering solutions to biology. I have contributed to the clinical development of multiple vaccines and biopharmaceuticals and recently published a book "Good Genes Gone Bad" by Penguin Publishers, on the lessons learned from failures in drug development. I worked as an assistant professor in gene therapy at the University of Pennsylvania, Philadelphia, and am on the NIH advisory committee for HIV vaccines

When did you begin work on gene therapy?

I started working on gene therapy in 1996 when I joined Jim Wilson at University of Pennsylvania.

Dr. Narendra Chirmule,


Why are there so few gene therapies currently available in the market?

The field of gene therapy has been through several ups and downs. The ability to induce stable, long-term gene expression, integrate appropriately into the host genome, and control of regulation are some of the challenges in the field. Each of these challenges requires extensive work and innovation.

What is the promise offered by viral vector gene therapies?

Viral Vectors currently are the most efficient way to transduce genes, utilizing their innate ability to infect, transduce, integrate exogenous genes. Immune responses to vectors are one of the major challenges in long-term use for the treatment of chronic diseases.

What are some of the innovative solutions being produced to overcome challenges in gene therapy clinical trials?

  • Adaptive clinical trial design
  • Targeted gene-editing technologies
  • Use of Multi-omics to analyze the multi-factorial effects on pharmacology, toxicology which translate to efficacy and safety
  • Selection of the most appropriate patient characteristics that have the highest likelihood to respond to treatments, through the use of machine-learning-based mathematical modeling, and many more.

Tell us about your professional background and your expertise

I am Professor of Medicine at University of California, San Diego and a cardiologist. I have been involved with gene transfer since 1993 and have initiated two FDA-sanctioned clinical gene transfer clinical trials with colleagues.

How will gene therapy for diabetes improve treatment?

Therapy for diabetes is complicated, which impedes compliance. If urocortin 2 gene transfer is as effective in humans as it is in rodents, it will make treatment easier. In animal models a single injection is effective for 2-years.

Dr. Kirk Hammond,

Professor of Medicine,
UC San Diego and Founder of
Renova Therapeutics

What are the unique requirements for a gene therapy clinical trial for diabetes?

a)The vector (AAV8) and transgene (urocortin 2) must be examined for biodistribution and toxicity in mice; b) a means to assess toxicity in the clinical trial must be established, with pre-specified surveillance intervals and rigorous safety monitoring; c) a dose-escalation clinical trial starting at low doses and gradually increasing dose, with intervals between dose groups to assess safety; d) the ideal study would be double-blinded, randomized and placebo-controlled and sufficiently powered to identify a dose or doses that would likely be safe and effective in larger clinical trials.

Is there a possibility for Type 1 Diabetes to be cured using gene therapy?

Cure is a word I would not use, in that clinical Type 1 diabetes involves immune destruction of beta cells, a condition that cannot be reversed (although islet transplantation can partially restore insulin production). However, because most Type 1 diabetes patients have insulin resistance, we have found that urocortin 2 gene transfer, which increases insulin sensitivity and insulin release, is effective in animal models of insulin deficiency, normalizing glucose homeostasis, reducing retinopathy and increasing survival.

What are some of the important genes being targeted for gene therapy for diabetes?

Urocortin 2, Insulin; Insulin plus Glucokinase; Insulin-like Growth Factor-1; Glucose-6-Phosphatase; NK1 fragment of Hepatocyte Growth Factor with Glucagon-like Peptide-1

Live Webinar on Risk Mitigation in Gene Therapy Clinical Development

Date: 09 December, 2021 | 10:00 – 11:00 EST

Gene Therapy is taking significant strides towards the market and therapy. Join our webinar on Risk Mitigation in Gene Therapy Clinical Development by our subject matter expert Dr. Atul Gupta, together with Dr. Narendra Chirmule and Dr. Kirk Hammond as they look at some of the barriers that are affecting gene therapy clinical trials, and the ways in which such challenges can be overcome.

Don’t miss the moderated thought-provoking panel discussions.

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