STOP CODON DISEASE
ACROSS ALL DISEASES, 30 MILLION PATIENTS CAN BE CLASSIFIED AS HAVING STOP CODON DISEASE
In 10 percent of genetically driven diseases, both rare and common, a single base pair of DNA is mutated, changing the code for an amino acid to a stop signal and causing a truncated or absent protein. These types of mutations, called nonsense mutations, can happen anywhere in the genome. If it occurs in the gene for dystrophin, it can cause Duchenne muscular dystrophy, but if it occurs in the gene for Factor VIII, it can cause hemophilia. As a result, the same exact mutation can be the cause of multiple diverse diseases.
PATIENTS ACROSS A NUMBER OF DISEASES COULD BE TREATED WITH A SINGLE tRNA MEDICINE, ACCELERATING AND SCALING THE DEVELOPMENT OF NEW GENETIC MEDICINES.
Alltrna is developing engineered tRNA medicines to readthrough premature termination codons and restore production of affected proteins, no matter what protein is affected.
A tRNA medicine that could universally treat patients across multiple indications in Stop Codon Disease could accelerate and scale the development of potential disease-modifying medicines for millions of patients, in a way that no other genetic medicine can.
POWERING BASKET CLINICAL TRIALS TO ACCELERATE THE CLINICAL DEVELOPMENT OF TREATMENTS FOR STOP CODON DISEASE
Basket trials bring together patients with different diseases into a single clinical trial. They test a treatment specific to a mutation, rather than to a gene. This approach has been used widely in oncology, resulting in regulatory approvals of breakthrough cancer treatments.
In the case of Stop Codon Disease, basket clinical trials will be key to scaling treatment for patients. Alltrna will initially focus on genetic liver diseases, and will select from among the 400 different rare and ultra-rare diseases in this category to design a trial with regulatory authorities.
“Through genetic testing, we learned that a specific mutation, called a premature termination codon, is causing our child's Duchenne muscular dystrophy. Learning that this mutation is shared across many diseases and that new medicines could be developed for this mutation gives us a sense of optimism for the future.”
PATIENTS CAN FIND OUT MORE ABOUT THEIR MUTATION STATUS BY ASKING THEIR SPECIALIST OR MEETING WITH A GENETICIST.
30 million people across thousands of diseases have a nonsense mutation that causes their disease.
Watch this video to learn more about genetic testing.
STOP CODON DISEASE IS JUST ONE OF THE THERAPEUTIC SPACES WE CAN TARGET WITH tRNA.
At Alltrna, we view tRNAs like no one else has, and we're on a mission to bring their unlimited therapeutic potential to millions of patients.
