The Gene Therapy Development Upgrade for the AI Era

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Gene therapy development is one of the most promising areas of innovation within the biotech industry. But new therapies face significant challenges at many steps along the path from discovery to clinic.
Form Bio’s FORMsightAI technology powers tailored in silico programs to help you make better therapies faster.
Connect with us to learn more and see what FORMsightAI can do for your therapeutic pipeline
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Biological Validation Studies
AI Architecture & Validation report pages

Biological Validation Studies

Explore our most recent lab results, demonstrating how FORMsightAI predicts and optimizes vector design outcomes.
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We collaborate with your team to build custom in silico programs to accelerate development.

Example Program Elements

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Drug Product Characterization

Detailed analysis of manufactured drug product, including proportion of full viral genomes to non-therapeutic packaged species.

Drug Product Characterization illustration

Assess the quality of bioreactor runs, identify unknowns

Determine proportion of full, partial and empty capsids from bioreactor samples

Analyze sequence variants and alignment to reference sequences

FORMsightAI solves your replication & packaging challenges to mitigate manufacturing risk.

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Multi-Candidate Comparison

Rapidly evaluate hundreds of millions of construct combinations in silico, comparing the safety, yield and expression of multiple candidates with different regulatory elements.

Compare combinations of promoters, ITRs, introns, backbones, RepCap, polyA elements and more

Rank constructs for triple transfection, CpG islands, GC content, transcription and translation, and manufacturability

Design for improved safety, efficacy and manufacturability and evaluate the impact of substituting regulatory elements

Multi-candidate comparison enables you to integrate large scale in silico modeling into your existing R&D process to hone in on the drug candidate most likely to succeed in the lab.

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Bioreactor Simulation

Test development processes in silico for production output in a virtual suspension-cell bioreactor.

Bioreactor Simulation illustration

Simulate the impact of altering triple transfection molar ratios, cell volume, HEK cell density or harvest times

Test and predict the impact of process changes on cell titers, empty/fill capsids and other CQAs

Save hundreds of thousands of dollars on test runs by optimizing process parameters in silico

FORMsightAI enables cell and gene therapy developers to simulate manufacturing output before spending the first dollar on bioreactor runs.

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Gene Expression Prediction

Analyze of mRNA for transcription and translation efficiency to predict the levels of therapeutic protein production.

Predict transgene expression in normalized transcripts 
per million vg

Understand impacts of regulatory element substitutions 
on mRNA protein expression

Model test lab mRNA degradation half life

FORMsightAI you can validate your expression model by comparing with qPCR and chemiluminescence data and easily use RNAseq is used for data analysis of the samples.

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Immunotoxicity Analysis

Formation of CpG islands is a reported trigger mechanism for immune response to many therapies. Form helps you identify CpG islands and score associated risks of potential immunotoxicity such as empty and partial capsids.

Immunotoxicity Scoring illustration

Score potential immunotoxicity risks associated with CpG islands, empty and partially filled capsids and other viral genomic patterns.

Analyze GC content and determine sites for CpG formation

Leverage powerful AI/ML models to predict propensity of full, empty and partial capsids

FORMsightAI enables your time to identify possible immunotoxicity effects and likelihood when making key changes to therapeutic construct design.

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AI-Powered Candidate Optimization

For a comprehensive and holistic optimization of construct for safety, efficacy and manufacturability engage with Form Bio to use our AI/ML expert services to optimize the CDS region.

Optimize CDS region of your construct for lowest truncation propensity and highest manufacturability

Optimize construct for gene expression and immunogenicity

Create derivative IP with our generative LLM AI models for identifying optimal versions of your starting construct.

By using FORMsightAI’s sophisticated models trained on vast public and private datasets, therapeutic developers can dramatically reduce the time and cost of drug development.

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Our Solution at a Glance
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More About Our Capabilities

Product Characterization

Fully understand constructs with automated AAV characterization reporting.

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Biomanfuacturing Simulation

Predict manufacturing results relating to truncations and other issues.

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Comparative Analysis

Compare an array of
constructs with different design characteristics to find the most promising versions.

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Generative Optimization

Enhance constructs to generate de novo designs with the best predicted yield, efficacy and safety characteristics.

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FORMsightAI models undergo continuous validation with biological studies conducted with independent lab partners and CDMOs.

benefits by the billions

Benefits by the Billions

Replication and packaging issues in cell and gene therapy development represent a multi-billion dollar industry-level problem. With FormSightAI, gene therapy companies can now tackle this challenge.

Truncation proportion: before and after

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Example: Predict truncation propensity and compare with predicted truncations from AI-optimized constructs

Reduce manufacturing costs

by avoiding costly bioreactor runs on suboptimal construct designs

Shorten time-to-market

by minimizing design inefficiencies that prolong manufacturing trial-and-error

Enhance safety

by predicting and optimizing capsid fill proportion with therapeutic drug product to streamline production scaling

Extend IP protection

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.

Jenny Marlowe
Jenny Marlowe,
Chief Scientific Officer,
Solid Biosciences

Solving the replication and packaging problem for gene therapy manufacturing

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.

These issues have significant implications for every gene therapy company:

  • 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.

  • 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.

Solving the replication and packaging problem for manufacturing

Stay Safe,
Stay Compliant

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.

*Source: Proposed DRAFT Guidance for FDA Consideration: Testing of Adeno-Associated Viral (AAV) Vector-Based Human Gene Therapy Products for Empty Capsids During Product Manufacture, 2021

Common Safety Risks and Challenges of Empty and Partially Filled Capsids

Empty and partially filled capsids compete with filled capsids for the same cellular receptors
Potential to trigger deleterious immune response
Reduced therapeutic dose yield, which decreases efficacy
Increased likelihood of adverse effects from binding to unintended targets
Increased variability of the resulting therapeutic product

How FORMsightAI Helps You Stay Safe

  • More therapeutic drug product

    We help gene therapy developers avoid the truncations and other issues that frequently lead to contaminated capsids.
  • Pre-emptive Compliance readiness

    Based on draft guidance, it is expected that the FDA will require gene therapy developers to demonstrate no more than 30% non-therapeutic capsids and no less than 70% full genome capsids for AAV therapies; our solution is designed to help gene therapies meet or beat that standard.
  • Fuller Capsids, Bigger Yields

    By reducing truncations and contaminants, and increasing full capsids by healthy double-digit percentages, we help increase the number of viable doses per manufacturing run.
  • Save Months and Millions

    We reduce the number of bench iterations, manufacturing process optimizations, and costs needed to reach clinical trial stages.
Stay Safe, Stay Compliant

Learn even more about our AI

Machine Learning for Cell and Gene Therapy

By Joe Nipko, Vice President of AI
Download Now
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Potential Economic Impact

Reducing truncations can favorably influence costs, yields and patient access
This illustrative example shows how significant the impacts of minimizing truncations can be for cell and gene therapy manufacturing economics.
example assumptions avg cost per bioreactor run table
Reduce cost per dose by more than
Increase manufacturing dose yields by
Treat a patient population of 50,000 in
5 Years instead of 10
Explore the Economic Impact of FORMsightAI.
Download the Worksheet
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Get Your Therapeutic Candidate to Market Faster

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