REPLIGENE LIFE SCIENCES LLP

In their own words

REPLIGENE LIFE SCIENCES THE MARKET: The overall cell and gene therapy market is growing rapidly and is predicted to witness an absolute growth of ~270% in the next 5 years with a compounded annual growth rate (CAGR) of ~25%. Applications have diverged from initial regenerative approaches using phage vectors to potential use of cell engineering for e.g. T cell modification using recombinant viral vectors for immunotherapy applications, and organ specific viral vector delivery. Currently over 1500 clinical studies are being conducted on gene therapy worldwide and the USFDA is anticipating more than 200 IND applications for cell and gene therapy products over the next few years. DEMAND ANALYSIS: To assess the health of the demand and supply dynamics of cell and gene therapy starting materials – plasmids and viral vectors, we performed an in-depth capacity analysis and generated a comprehensive data-based model to project plasmid and viral vector manufacturing needs and corresponding global production capacity at big pharma and CDMOs (Contract Development and Manufacturing Organization). Even after accounting for all the announced global capacity expansions at CDMOs there is a gap of roughly 30-40% between the demand vs. the supply of cell and gene therapy adducts. THE PROBLEM: Just the way an Active Pharmaceutical Ingredient (API) lies at the core of a traditional pharmaceutical, at the core of all cell and gene therapy lie plasmids and vectors. For any gene modification, whether it is for cell engineering or for viral vector production, one has to start by generating a plasmid with the mutation/gene of interest. Viral vectors are used to manipulate and correct host DNA in gene therapy. Adeno-associated virus (AAV) and Lentivirus (LV) vectors have emerged as the leading viral delivery modalities for gene therapy. AAV vector generation utilizes a triple plasmid transfection where the three plasmids include pGOI, pRepXCapY and pHelper. Similarly for LV, four plasmids are required, pGOI, pGagPol, pRev and pEnvelope. While the pharmaceutical industry has achieved top tier efficiency in the production of chemical moieties (API) and biologics (e.g. proteins, antibodies, interferons and vaccines), manufacturing of plasmids and viral vectors has fallen through the cracks; likely because their process of manufacturing differs significantly from traditional biologics. The development of standardized production platforms, tools, and specialized starting materials has been hindered by the unique nature of the production process of cell and gene therapy adducts, which also requires high upfront investment. This has had an adverse impact on the market in three ways 1. LOW SUPPLY: Low yields of production couple with a technically and financially challenging process of setting up scale-up manufacturing facilities for these adducts that has led to a significant supply demand imbalance for high quality supercoiled plasmids and high titre viral vector constructs 2. LAG TIMES: Current waiting time at several leading US suppliers of plasmids and viral vectors is estimated to be 12-18 months. This further supports our data analysis that pointed to a huge supply and demand imbalance for these products. 3. AFFORDABILITY: With select few suppliers across the world, supply demand imbalance and production complexity has driven the cost of cell and gene therapy adducts to exorbitant levels, which in turn has made cell and gene therapy development cost prohibitive in India and practically inaccessible for large populations of the world including India. Given the high demand of viral vectors and plasmids in milligram to gram quantities; fungible, optimized manufacturing and purification platforms are the need of the hour. INNOVATION In the first phase of our project we have focused on production of high quality supercoiled plasmid DNA and viral vectors that are used most commonly in the clinic. We have developed a platform suite of technologies that allows us to rapidly iterate and build scalable protocols for different types of plasmids at an affordable price while maintaining high quality and purity of plasmids on a lab scale. The science behind our innovation targets the most crucial steps in manufacturing process and design platforms across the core three aspects of plasmid and vector manufacturing 1) We have developed a codon optimization protocol that helps us generate high yield plasmid and minimize sequence mutations and deletions that are the most common drivers of low yield and expression of incorrect sequences. Our codon-optimized plasmid has been demonstrated to give a high viral titre and shows high expression when compared to regular plasmids in standard expression cell lines. In addition, our codon optimization data library and yield maximizing processes can be applied to any type of plasmid or viral vector. 2) We have developed a series of iterative protocols around scaled up production of plasmids

Written by the company on its public Startup India profile. Not verified by us.

Is this your company?

Everything here comes from public government records. If something is wrong, the authoritative place to correct it is the Startup India portal itself, and we will pick the correction up on the next monthly snapshot. To flag an error on our side, use the report link or write to trust@indianstartupmap.com.

How we built this record Where the data comes from How you may reuse it