Cell & Gene Therapies (CGTs) are a transformative class of medicines that aim to treat, prevent, or potentially provide curative therapies to diseases that could not be cured by traditional medicines.
While gene therapy involves the transfer of genetic material, usually in a carrier or vector, and the uptake of the gene into the appropriate cells of the body, Cell therapy involves the transfer of cells with the relevant function into the patient. Both approaches have the potential to alleviate the underlying cause of genetic and acquired diseases. CGTs in various forms has produced clinical benefits in patients with blindness, neuromuscular disease, haemophilia, immunodeficiencies, and cancer etc.
Like with any drugs, CGTs also need to undergo clinical trials, to prove their efficacy and safety. In general, there are three phases to each clinical trials, namely, Phase 1, Phase 2 and Phase 3, each involving a slightly greater number of people. In case of CGTs, Phase 1 and 2 are often combined to accelerate the time of development or help decrease costs. They are also combined in case the disease being studied is rare and the number of patients living with it very small. Phase 3 clinical trial, conducted in larger group to compare the efficacy and safety of new therapy with the current treatments, tends to be the longest and is usually the basis for regulatory approvals. Conducting clinical trials for CGTs are more difficult than traditional pharmaceutical trials because the nature of risks associated with CGTs are much higher- disease are either rare or severe, the number of patients might be limited, side-effects could be long-term, severe and unexpected, and the investment is much higher.
At CRNI we understands the challenges these studies present compared to other traditional clinical trials. CRNI has network of experienced multi-disciplinary CGT experts and centres who are passionate to help bring life changing treatments to patients. We work extensively with pharma and biotech customers to craft tailored solutions to help sponsors optimize and accelerate the clinical development aspect of CGTs. One of the key challenges faced in conducting CGT clinical trials is recruiting patients, especially when the CGT is targeting a disorder which either rare, paediatric related or at advanced stage. CRNI uses patient- centred approach to manage enrolled patients because their safety is the first priority. We are committed to design study protocols with accelerated subject recruitments. We closely collaborate with regulatory agencies to ensure compliance and faster study outcomes.
Copyright © 2022 Clinical Research Network India All Rights Reserved by