Envisioning a new future for the treatment of rare diseases

Envisioning a new future for the treatment of rare diseases

Our proprietary chemistry platform enables the precise design, optimization and production of stereopure oligonucleotides.

This may allow for optimized pharmacological properties, including greater stability, enhanced activity and selectivity, and longer duration of effect, and has broad applicability across modalities including antisense, exon-skipping, microRNA, RNAi and RNA-guided gene editing. The improved pharmacologic profile opens up the potential for more effective medicines for many diseases for which there are currently few or no treatment options.

Our programs in

Huntington’s disease (HD) is a fatal genetic disorder that causes the progressive loss of neurons in the brain, resulting in cognitive decline, psychiatric illness and chorea.

HD is an autosomal dominant disorder in which each patient has inherited one mutant huntingtin (HTT) allele and one wild-type (healthy) HTT allele that is critical for neuronal function.

An expanded CAG triplet repeat in the HTT gene results in the production of mutant HTT (mHTT) protein, leading to the loss of neurons in the brain.

Our investigational stereopure oligonucleotides target single nucleotide polymorphisms (SNPs) associated with the expanded CAG repeat in the mHTT gene. The presence of specific SNPs is used to identify and locate the mutant allele, with the objective of silencing only the disease-causing mHTT gene transcript while leaving the healthy allele relatively intact.

The C9orf72 gene provides instructions for making protein found in various tissues, with abundance in nerve cells in the cerebral cortex and motor neurons. Mutations in the C9orf72 gene are the strongest genetic risk factor found to date for both amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).

In both diseases, pathogenic transcripts of the C9orf72 gene contain hundreds to thousands of hexanucleotide repeats, compared to 2-23 in wild-type transcripts.

Our optimized investigational stereopure antisense oligonucleotide aims to selectively silence the repeat-containing transcripts in C9orf72.

  • Huntington’s
    disease

    Huntington’s disease (HD) is a fatal genetic disorder that causes the progressive loss of neurons in the brain, resulting in cognitive decline, psychiatric illness and chorea.

    HD is an autosomal dominant disorder in which each patient has inherited one mutant huntingtin (HTT) allele and one wild-type (healthy) HTT allele that is critical for neuronal function.

    An expanded CAG triplet repeat in the HTT gene results in the production of mutant HTT (mHTT) protein, leading to the loss of neurons in the brain.

    Our investigational stereopure oligonucleotides target single nucleotide polymorphisms (SNPs) associated with the expanded CAG repeat in the mHTT gene. The presence of specific SNPs is used to identify and locate the mutant allele, with the objective of silencing only the disease-causing mHTT gene transcript while leaving the healthy allele relatively intact.

  • Amyotrophic lateral sclerosis
    and Frontotemporal dementia

    The C9orf72 gene provides instructions for making protein found in various tissues, with abundance in nerve cells in the cerebral cortex and motor neurons. Mutations in the C9orf72 gene are the strongest genetic risk factor found to date for both amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).

    In both diseases, pathogenic transcripts of the C9orf72 gene contain hundreds to thousands of hexanucleotide repeats, compared to 2-23 in wild-type transcripts.

    Our optimized investigational stereopure antisense oligonucleotide aims to selectively silence the repeat-containing transcripts in C9orf72.

Emerging therapeutic areas

Our goal is to develop transformational therapies for conditions for which there are currently few or no treatment options. While our initial focus is in neurology, our stereopure chemistry platform may unlock the potential for transformative therapies in many therapeutic areas. We currently partner with Pfizer in the hepatic space, for example, and have demonstrated compelling pre-clinical data in other areas including ophthalmology and dermatology.

Our diverse discovery programs aim to explore the potential of our platform across a broad range of serious genetic diseases.

Explore our pipeline

Emerging therapeutic areas

Our goal is to develop transformational therapies for conditions for which there are currently few or no treatment options. While our initial focus is in neurology, our stereopure chemistry platform may unlock the potential for transformative therapies in many therapeutic areas. We currently partner with Pfizer in the hepatic space, for example, and have demonstrated compelling pre-clinical data in other areas including ophthalmology and dermatology.

Our diverse discovery programs aim to explore the potential of our platform across a broad range of serious genetic diseases.

Explore our pipeline