Question 1

What is Ascidian Therapeutics?

Accepted Answer

Ascidian Therapeutics is a company that is redefining the treatment of disease by rewriting RNA. They were inspired by the way ascidians, also known as sea squirts, re-engineer their transcriptome through RNA trans-splicing and alternative splicing during their growth from larvae to adults.

Question 2

What is Ascidian Therapeutics' technology platform?

Accepted Answer

Ascidian Therapeutics' first-of-its-kind RNA exon editing platform is designed to expand the therapeutic possibilities of RNA medicine and treat diseases not addressed by today's gene editing technologies. By rewriting RNA, Ascidian's exon editing technology provides the durability of gene therapy without the risks of direct DNA editing or gene replacement, while maintaining endogenous gene expression patterns and levels.

Question 3

How does Ascidian Therapeutics' approach differ from other RNA therapeutics?

Accepted Answer

Ascidian Therapeutics' approach differs from other RNA therapeutics in that it can replace multiple contiguous exons, not just single bases. This provides a more powerful and versatile RNA therapeutic approach, as disease-causing genetic mutations are often located at different sites on the gene in different patients.

Question 4

What are the potential benefits of Ascidian Therapeutics' RNA exon editing technology?

Accepted Answer

Ascidian's RNA exon editing technology enables precise post-transcriptional editing of genes, resulting in full-length, functional proteins at the right levels in the right cells at the right time. This approach has the potential to revolutionize the treatment of human disease by addressing the underlying causes of disease.

Question 5

What is Ascidian Therapeutics' vision for the future?

Accepted Answer

Ascidian Therapeutics' vision is to create a new class of medicines that address the underlying causes of disease by rewriting RNA. They are leveraging cutting-edge genomics, computational biology, and deep-sequencing technologies to develop innovative RNA-based therapies that can provide a sea change in the treatment of human disease.

Ascidian Therapeutics

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Frequently Asked Questions about Ascidian Therapeutics

What is Ascidian Therapeutics?

What is Ascidian Therapeutics' technology platform?

How does Ascidian Therapeutics' approach differ from other RNA therapeutics?

What are the potential benefits of Ascidian Therapeutics' RNA exon editing technology?

What is Ascidian Therapeutics' vision for the future?

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