Submitted Genes
Highly-targeted
gene specific programs
A personalized mutation-driven drug discovery program
Our nano-rare patients are a very, very small patient population, often many will be the only one in the world with their specific gene mutation. As such, the majority of our patients have a unique-to-them drug discovery program in development.
Our genes are large and there can be many different mutations within a single gene. Each mutation can cause a very different outcome in terms of cause of disease, disease symptoms and severity. At n-Lorem, we not only target the specific gene, but design our program to address the specific gene mutation for each patient.
It is our hope that, as more genome sequencing is conducted, more patients will be identified that have a gene mutation that fits a program we are already working on. For those patients, we will already be well ahead in discovering and developing a potential experimental ASO medicine.
Submitted genes
While we cannot accept every application that is submitted to n-Lorem, we learn from each application and continue to advance our understanding of nano-rare patients and their unique illnesses.
Nano-rare patients present a unique opportunity to learn about health and disease, and we are obligated to learn maximally from each patient and our aggregate experience and share our learnings with all interested parties as rapidly as possible.
Below is a list of all the genes that have been submitted to n-Lorem. This list is updated quarterly. In some instances, we have had multiple submissions for a single gene, each submission with different gene mutations.
What we cannot treat today:
- Truncating variants
- True null or loss of function variants
- Homozygous gene mutation
However, ASO technology continues to advance at a rapid rate, and we hope to one day be able to broaden our efforts to include nano-rare patients who are not amenable to ASO technology today.
Genes from all submissions:
Not all are active programs
- NOTE:An orange dot next to a gene indicates an active program or an available ASO therapy. Click on the gene name to learn more.
What do I do if my gene isn’t listed?
This is a list of all of the genes that have been submitted to n-Lorem. Not all are active programs. To learn more if you or your patient qualifies for n-Lorem please visit one of the following links:
We cannot do
this alone
Together we are changing the world—
one patient at a time
We hope that you join us on this journey to discover, develop and provide individualized antisense medicines for free for life for nano-rare patients. The ultimate personalized medicine approach – for free, for life.
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