The Cell and Gene Therapy Design Upgrade for the AI Era
Deficiencies in replication and packaging of genomes into assembled capsids cause manufacturing efficiency and therapeutic yield to plunge.
These inefficiencies account for millions of dollars in capital and months/years of manufacturing trial and error per pipeline candidate.
Solving for replication and packaging issues in construct design addresses a multi-billion dollar problem for the cell and gene therapy industry.
Running a therapeutic construct through a bioreactor can be highly unpredictable. This guessing game can cost CGTs millions in manufacturing costs and months or years in time-to-market.
Cell and gene therapy companies, more than ever, can’t afford to waste time and capital on manufacturing scale up trial and error.
Failure to optimize therapeutic design for manufacturing can result in lower yields and higher costs-per-dose, making therapies less accessible for patients and squeezing margins for CGT companies.
Predict your construct’s manufacturing output before spending up to millions of dollars on bioreactor test runs.
With FORMsightAI SIMULATE you can:
Predict non-empty/empty capsid ratios
Predict capsid content proportions by full, truncated and chimeric components
Predict truncation propensities
Breakdown truncation contribution proportions by sequence region
Generate new construct derivatives with the greatest predicted likelihood of manufacturing success and cost efficiency.
With FORMsightAI OPTIMIZE you can:
Generate the ideal construct for maximized predicted therapeutic drug products produced while minimizing key inefficiencies
Develop a new construct design blueprint
Our experienced team helps yours customize, enhance and operationalize your cell and gene therapy manufacturing program. Meet one of our team members, Alpha Diallo, PhD:
by avoiding costly bioreactor runs on suboptimal construct designs
by minimizing design inefficiencies that prolong manufacturing trial-and-error
by predicting and optimizing capsid fill proportion with therapeutic drug product to streamline production scaling
by filing fresh patents on optimized construct derivatives generated with FORMsightAI
As a Form Bio customer, we are thrilled with the uniquely valuable AI-driven insights they’ve contributed to our research and development efforts. By working closely with this world class team and cutting edge technology, we can clearly see that applying innovative AI solutions to pre-clinical cell and gene therapy development offers exciting potential for CGT companies’ economics, market timing and ability to impact human health.
In 2021, the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) shared a report and draft guidance* on safety issues for AAV-based gene therapies. In its report and FDA draft guidance, CTGTAC singled out the importance of screening AAV-based gene therapies for empty and partially filled.