Harnessing the
human system for
Gene Therapy.

ShapeTX is developing a new generation of Gene Therapies for some of the most daunting genetic diseases in the world. Our proprietary RNA technologies leverage machinery already present in human cells to safely and efficiently correct genetic mutations, while avoiding immunogenicity and DNA damage. This process will provide safer treatments that work with the body, not against it. We believe the cure is in you, we let biology reveal the way.

Scroll To Learn More

Expressing our true genetic code.

Mutations in DNA are carried into mutated RNA which changes how a single letter is translated into a protein. A misfire caused by a single DNA letter change can lead to dysfunctional or toxic protein products.

Many upcoming gene editing approaches, like CRISPR or Base Editing, rely on using foreign proteins to initiate genetic changes in the cell. However, our immune system evolved to specifically recognize, target, and eliminate cells harboring foreign components. Therapies rejected by the body’s immune response lose efficacy and can cause severe toxicity—even death. Our solution is to avoid immunogenicity and DNA alterations altogether by leveraging naturally occurring editing systems already present in our cells. The proprietary therapeutics we’ve created allow cells to read the correct genetic code and express the right protein, thereby fixing the disease.

water2

Payload – RNA FixTM

For diseases generated by point mutations, we employ RNAfix™, a fit-for-purpose modified gRNA (guide RNA) that’s designed to recruit ADAR—a naturally occurring enzyme editor found in all human cells—to change how a single letter is read and subsequently expressed as a protein. Enabling a targeted RNA edit using ADAR can serve to correct mutations or purposefully create them to treat a vast array of genetic disorders. RNAfix™ can be used to induce A-to-G correction of point mutations, knockdown protein expression, induce exon-skipping, as well as modulate protein-protein interactions. RNAfix™ payloads can be gene encoded into AAV vectors or administered directly as chemically-modified RNAs, enabling flexible delivery and optimal efficiency. ShapeTX is currently pursuing both delivery approaches in various indications.

Payload – RNA SkipTM

For diseases caused by premature stop codon mutations, we employ RNAskip™, a proprietary suppressor tRNA specifically engineered to recognize premature stop codons and enable translational readthrough to produce a fully corrected protein. There are only three genetically-possible premature stop codon sequences, making our suppressor tRNA therapy re-deployable across genetics disorders.1 RNAskip™ payloads can be gene encoded into AAV enabling long term duration of our therapy.

Delivery – AAVidTM

Innovative payloads require innovative delivery tools. Currently, many available Adeno Associated Viruses (AAVs) across the industry are plagued with severe limitations. From low tissue specificity, pre-immune exposure to poor manufacturing, AAV needs to be as advanced as the payload it carries.

 

To address delivery challenges, we built our AAVid™ capsid discovery platform that yields industry-leading, next-generation AAVs with optimized delivery properties. Equal parts molecular engineering and data science, our purpose-built AAV vectors can be used to efficiently deliver both our RNAfix™ and RNAskip™ payloads—as well as other industry leading payloads—to disease specific tissues. By combining rational mutational targeting with cutting-edge DNA synthesis technologies, we’ve generated massive candidate capsid libraries with unique AAV variants in the billions per single campaign.

Manufacturing

Blending our expertise in genetic engineering, cellular therapy, and biologics manufacturing, we’re reshaping what AAV manufacturing means for the distribution of life-changing drugs. Using a proprietary human stable cell line production technology, we’re rebuilding AAV manufacturing to break down the barriers that prevent gene therapies from reaching those who need them most. The final products are drastically improved in yield and quality as well as higher in culture density, meaning our RNA therapies do more with less. Our manufacturing platform will enable us to accelerate drug innovation, reduce common toxicities, and result in increased accessibility through reduced scale and cost of production.

A sustainable end-to-end RNA therapy platform for the world’s most challenging genetic diseases.

  • list-1
    Versatility
    Several mutation types result from base substitutions, so our RNA therapies have wide coverage and application over thousands of diseases.
  • list-2
    Biocompatibility
    By harnessing human systems from within, our therapies minimize the risk of immunogenicity and DNA damage, producing results in intractable diseases.
  • Everlasting
    Our payloads stay in the cell as long as the cells are alive, enabling one-time, long-term—if not permanent—treatment.
  • Sustainability
    Efficient manufacturing enables higher-yield and lower dosage requirements, making Gene Therapy economically feasible over time.
  • Innovation + Data
    Results bring data, and data drives our decision-making process toward the development of new therapies.
Pipeline
Swipe left to learn more
Gene Therapy advances when data meets imagination.

Others carved the path for where we are today, but we carry the torch to new terrains. RNA targeting is at the forefront of a radically new way to treat a range of diseases. And we’re just getting started.

1958

Discovery of tRNA

1965-66

Discovery of AAV

1965

Identification of suppressor tRNAs

1987

CRISPR RNAs first described

1987

ADAR first described

1995

ADAR as a therapeutic POC

1996

First clinical trial using AAV

2000-2002

First in vivo suppressor tRNA study

2012

Programmable CRISPR Cas9. Congrats on the Nobel!

2013

ADAR-oligo fusion POC

2013

CRISPR-Cas9 editing with in human cells

2016

First ex vivo CRISPR edited cells used to treat lung cancer

2017

First AAV encoded ADAR editing in vivo.

2018

ShapeTX is born

2019

First endogenous ADAR editing using genetically encoded guide RNA only.

Swipe Left To Learn More