Gene & Cell Therapy

Advancements in gene and cell therapy with innovative science and technology approaches

A new era in gene therapy approaches and applications

Through a gradual evolution spanning more than two decades, gene and cell therapies have undergone significant advancements. This has led to remarkable improvements in these treatments’ precision, efficacy, and safety. The FDA and EMA have approved more therapies in the last year, including Eticel™, Valrox™, and Hemgenix™, than any previous year, indicating significant progress in the field.

CRISPR CAS9, ZFN (Zinc finger nucleases) and other gene editing technologies have gained significant spotlight, they are still far from receiving widespread regulatory approvals. While approvals are less frequent for gene therapies, thousands of clinical trials are currently in progress to test these novel therapeutics. Specifically, advancements focused on addressing the following challenges:

Delivery and distribution

Targeting and tropism

Gene expression and regulation

Vector safety and immunogenicity

Generations of Gene Therapies

Expanding the scope of gene therapy for complex diseases

The field of gene therapies has undergone a significant transformation since its inception. What began as a focus on targeting monogenic mendelian diseases has evolved to address complex polygenic genetic diseases. This progress has been marked by a shift from empirical approaches to deep sciences and generative AI, enabling the design of more efficient and effective experiments. Key advancements in gene therapies and current progress highlights:

Target tissue specificity

Tropism and targeting have evolved to achieve exceptional specificity for cells, tissues, and organoids, with further advancements in precision and timing controls continuously emerging

De risking gene therapies

Safety and immunogenicity studies yielded valuable insights into reducing associated risks of gene therapies by addressing specific challenges such as vector-host integration, immune responses, and off-target effects

Viral & Non-viral vector design & optimization

As viral and non-viral vectors evolve, a better understanding of fit for purpose vectors and novel capsid designs enables progress beyond traditional AAV, Adeno, Herpes Simplex, and Lentiviruses to innovative options like anellovirus and more

Next-generation technologies

Next-generation technologies in gene expression and regulation enable better matching of patient needs, allowing for smaller doses with higher protein expression in specific tissues rather than systemic delivery expression

Revolutionizing gene therapy with AI and innovative approaches

Advancements in machine learning, artificial intelligence (AI), and high-throughput sciences have contributed significantly to the rapid progress in gene therapy. By combining cutting edge techniques from various fields, researchers are unlocking the full potential of gene therapies for a wide range of diseases

In silico capabilities are accelerating the development of gene cell therapies and newer modalities like mRNA and ASO by leveraging the combinatorial power of:

Single-cell RNA sequencing

Structure prediction

Computational chemistry

Structural biology

Aganitha’s Point of View

Precision Biology and Therapeutics for the Future

Our precision-focused strategy unlocks the full potential of gene therapy, empowering researchers to develop innovative treatments that match patient’s specific needs

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Emphasizing precision biology for better targeted therapeutics not only at the protein level but also at the tissue and organ levels
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Developing precision therapeutics with optimized genetic payloads and advanced generation treatment strategies (e.g., payloads that do not fit in one AAV capsid)
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Implementing targeted science through rigorously designed specialized animal studies, primate work, and clinical trial designs
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Focusing on precision not just for personalized medicine but also for creating a class of drugs matched to the specific needs of multiple patients, extending the benefits of precision medicine beyond individualized and costly treatments
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Augmenting lab work with innovative de novo capabilities, such as advanced vector design techniques, capsid optimization strategies, and in-depth toxicology analysis

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Employing precision matching for patient cohorts, biomarkers, disease studies, and regulatory submissions to optimize the efficacy and safety of gene therapies

Examples of solutions we have developed for our clients

Innovative use cases and cutting edge techniques in gene therapy

Enhanced tropism prediction

Our advanced in silico simulations allowed for the identification of viral vectors with higher efficacy in targeting specific tissue types, for example, targeting blood-brain barrier receptors

Optimized vector harvesting

Leveraged ML models to analyze published data on capsids that helped design improved versions with a greater affinity towards target tissues. We can extend the approach to optimize the manufacturing process

Comprehensive safety evaluation

Our viral vector integration studies helped to examine host site integrations, effects on surrounding genes, and potential genotoxicity, leading to a thorough assessment of safety and efficacy

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