Unleashing the programmable power of CRISPR genome editing via AI
Pioneering One-Off Curative CRISPR Solutions for Genetic Diseases
The story of CRISPR in medicine so far has been one of remarkable speed. It took just 11 years from the 2012 paper in which Innovative Genomics Institute (IGI) founder Jennifer Doudna and Emmanuelle Charpentier and colleagues first described CRISPR genome editing to reach the first CRISPR-based therapy approved by regulators in the UK and the US. However, leveraging the platform nature of Crispr has not yet been realised. Until now.
With generative AI platform processes will be built via the leveraging of AI in biotechnology, medicines can be designed using standardised rules. Manufacturing will be done by standardised rules. Distribution will be done using standardised rules. CRISPR IND’s and CMC’s will become standardised under AI-driven drug development. Testing – the most expensive part of IND programs – can be and will be done via AI-designed digital twins
CRISPR for Neurodegeneration and Heart Disease
Genuron is at the forefront of developing turnkey systems for engineering CRISPR therapeutics, aiming to transform the landscape of genetic medicine. By enabling precise edits to the human genome, we tackle the root causes of genetic disorders, offering hope to millions worldwide. Our mission is to make personalized therapies accessible and equitable, leveraging innovative regulatory strategies to ensure widespread distribution and affordability.
Alzheimers Disease
The brain is a primary target for Alzheimers medicines as the genetic targets for Alzheimer’s are already known. People with two copies of the faulty APOEb4 gene are 12 times as likely to develop the condition, studies suggest. Now that AAV’s are being developed that efficiently crosses the blood-brain barrier in human cell models and delivers genes throughout the brain in mice expressing the human protein, the potential for gene therapies for neuodegeneration are on the horizon.
Ischemic heart disease and stroke
Genuron is applying genome editing to the hundreds of devastating genetic diseases of the heart and muscle that currently do not have cures. Cardiac and skeletal muscle tissues are long-lasting, allowing for possible one time” fixes. By changing a handful of nucleotides in a base editing molecule, a heart disease gene therapy could be used for a variety of diseases while being manufactured locally in widely distributed facilities. Combined with universal newborn screening and routine testing of affected individuals, this approach could be a model for the worldwide treatment of genetic diseases.
Step-by-Step Guide to CRISPR editors
CRISPR nuclease
Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated (Cas) systems provide bacteria and archaea with adaptive immunity against viruses and plasmids by using CRISPR RNAs (crRNAs) to guide the silencing of invading nucleic acids. The CRISPR system consists of a short non-coding guide RNA (sgRNA) made up of a target complementary CRISPR RNA (crRNA) and an auxiliary transactivating crRNA (tracrRNA). The sgRNA guides the Cas9 endonuclease to a specific genomic locus via base pairing between the crRNA sequence and the target sequence, and cleaves the DNA to create a double-strand break.
Base editor
Base editing leverages the advanced DNA-scanning and sequence-identification features of the CRISPR-Cas9 system in conjunction with a deaminase enzyme, allowing for the introduction of single nucleotide polymorphisms by chemically modifying the target DNA sequence without deliberately creating a DNA double-strand break. This process, known as deamination, involves the removal of an amino group from a nucleotide, ultimately leading to the incorporation of a new base during DNA repair or replication.
Prime Editor
Prime editing is a “search and replace” gene editing method in which a reverse transcriptase (RT) is fused to the C terminus of Cas9 H840A nickase. The fusion enzyme is capable of installing targeted insertions, deletions, and point mutations using a prime editing guide RNA (pegRNA). As with a typical gRNA, the pegRNA is designed with a spacer that binds to a specific genomic DNA locus and directs the nickase to the target site. The longer pegRNA also encodes a primer binding site (PBS) and the desired edits on an RT template.
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