FORMsightAI

The Cell and Gene Therapy Design Upgrade for the AI Era

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Cell and gene therapy development is one of the most promising areas of innovation within the biotech industry. But new therapies face severe challenges at the manufacturing stage due to replication and packaging issues that result in lower therapeutic yields.
Form Bio's FORMsightAI is a breakthrough AI-powered solution for addressing the top issues that make biomanufacturing difficult, time-consuming and expensive.
Connect with us to learn more and see FORMsightAI in action
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Solving the replication and packaging problem for cell and 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 cell and 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.

  • 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.

Solving the replication and packaging problem for manufacturing

Our Solution at a Glance

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FORMsightAI SIMULATE

Predict your construct’s manufacturing output before spending up to millions of dollars on bioreactor test runs.

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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

Learn More About SIMULATE
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Broad Cell & Gene Therapy Use Cases

EXAMPLE
NUCLEIC ACIDS
DNA
siRNA
miRNA
mRNA
CRISPR Constructs
EXAMPLE
VECTORS
AAV
Adenovirus
Lentivirus
HSV
LNP
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Advanced AI Models

  • CPG ISLAND DEtection
    For enabling scaled usage of ASOs
  • Substitutional CODON OPtimization
    For identifying the best substitutions of chemically interchangeable amino acids
  • Hairpin Propensity Analysis
    For determining alternative nucleotide substitutions
  • Promoter OPtimization
    For elevating gene expression levels
  • Methylation Analysis
    For enhancing gene expression regulation

FORMsightAI OPTIMIZE

Generate new construct derivatives with the greatest predicted likelihood of manufacturing success and cost efficiency.

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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

Learn More About OPTIMIZE
Our Solution at a Glance

Expert Professional Services

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:

CASE STUDY

Case Study:
Publicly Traded
Gene Therapy
Company Elevates Manufacturability of Constructs with FORMsightAI

By partnering with Form Bio, the company’s manufacturing technology leaders discovered how innovative AI and machine learning models can be leveraged to reduce truncations that can lead to yield, safety and efficiency issues for pre-clinical therapies
Download the Case Study
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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, cell and 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

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

Whitepaper:
‍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
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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
45%
Increase manufacturing dose yields by
88%
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 Started Today

Bring the Next
Biotherapeutic to Market, Faster

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