Pipeline

We are developing a pipeline of novel, proprietary therapeutic products that target high-penetrance disease genes to alleviate and potentially cure genetic disorders in patients. Our pipeline includes candidates directed at validated targets: disease-causing mutations in the human genome that, if corrected, would lead to improvement and potential cure for patients with a wide variety of genetic disorders.

Our current pipeline is rich with potential:
 

  • Multiple development programs

  • Diverse target organs

  • In vivo administration

  • Addressing significant unmet medical need

 

Sickle Cell Disease

 

Sickle cell anemia is a disease that is hereditary – passed from one generation to the next. It affects people closer to their African origins – so, in the United States, it is more common in African-American (1 in 500 newborns) and Latino children (1 in 1,000 newborns). It is caused by a single letter in a person’s DNA, or genetic code, that normally makes red blood cells their proper, donut-like shape, and able to carry oxygen. A person with sickle cell disease makes red blood cells, but they are shaped like sickles, causing them to get stuck in blood vessels near the brain, kidney, lungs, and bones, causing very severe pain. These events start to happen to children very early and accumulate, causing damage to these organs – 1 in 10 patients with sickle cell have a stroke by the age of 20 – extremely young. This takes a very serious toll on patients – the average age of death is in the 40s.

 

There is treatment for sickle cell disease, called hydroxyurea, that is effective, but has some difficult side effects and must be taken daily for life. New medicines are being developed by several companies, for the first time in decades, that are starting to show a positive impact for patients in clinical studies.

It is possible to be cured of sickle cell disease, but the procedure – which is a bone marrow transplant – is risky and complex and is therefore only offered to patients with severe disease who meet many criteria to ensure success. There are several companies working on applying gene therapy and gene editing to try and come up with ways to cure sickle cell disease that are more safe and effective and therefore could be available to many more patients.

We’ve known the cause of sickle cell disease for over 60 years – a single letter in the genetic code in generations of certain families – and it is exciting to see that science has now matured enough to come up with more effective medicines, and hopefully soon, cures that could be available to many more people.

Podcast on Sickle Cell Disease: a mother’s perspective


The Tote and Pears Podcast - Episode 17: Sickle Cell
While many parents focus on juggling work, family and many other personal obligations, some women spend all of their energy praying for another day of life. In this episode, Monique Fritz shares her story of caring for her son, Cameron, who was born with sickle cell anemia.

Cystic Fibrosis

Cystic Fibrosis is also a hereditary disease – passed from one generation to the next. It primarily affects people of Northern European descent. It is caused by a change of one or a few letters in a person’s DNA, or genetic code, that impacts their cells ability to transport chloride, affecting secretions in the lungs and intestines that are required to maintain health. A person with cystic fibrosis has abnormal secretions in the lung, intestines, pancreas, and elsewhere – causing serious lung disease and digestive problems from an early agethe average age of death is currently in the 40’s.

There is now effective treatment for cystic fibrosis, but these must be taken daily for life. It is not yet possible to be cured of cystic fibrosis. We’ve known the genetic cause of this disease for over 30 years – a small change in the genetic code in generations of certain families. It’s time for a one-time practical cure for patients with this disease.

Future Platform Applications

We have the potential to leverage our proprietary chemistry technology into other areas of high therapeutic potential, including the use of PNAs as anti-sense oligonucleotides (ASOs) for gene regulation and the application of our fully synthetic system to next-generation CAR-T.

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