Advancing a new class of therapeutics through targeted protein upregulation.
Our RAP Platform® is enabling the discovery and development of potential therapies for a broad spectrum of genetic diseases. Using this pioneering approach, we can generate therapeutic candidates capable of specific, tunable and durable protein amplification.
We are exploring candidates in multiple indications with genetically validated targets, with an initial focus on central nervous system and metabolic diseases.
CMP-002 |
|
|---|---|
| Indication | SYNGAP1-related disorder |
| Target | SYNGAP1 |
| Anticipated Milestones | Clinical initiation in Q4 2026. Topline data 1H 2028. |
| Commercial Rights |  |
ProgressIn between Phase 1 / 2 |
|
SHANK3 |
|
|---|---|
| Indication | Phelan-McDermid Syndrome |
| Target | SHANK3 |
| Anticipated Milestones | Development Candidate in 2027. |
| Commercial Rights | |
Progressin discovery & preclinical phase |
|
CNS Program 1 |
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|---|---|
| Indication | CNS haploinsufficiency |
| Target | |
| Anticipated Milestones | |
| Commercial Rights | |
Progressin discovery & preclinical phase |
|
CNS Program 2 |
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|---|---|
| Indication | CNS haploinsufficiency |
| Target | |
| Anticipated Milestones | |
| Commercial Rights | |
Progressin discovery & preclinical phase |
|
CMP-001 |
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|---|---|
| Indication | Urea Cycle Disorders |
| Target | CPS1 |
| Anticipated Milestones | |
| Commercial Rights | |
Progressin phase 1/2 Exploring potential partnership opportunities. |
|
| Strategic research collaboration to identify and develop antisense oligonucleotide (ASO) drug candidates for multiple gene targets relevant to neurodegenerative and kidney disease indications. | |
|---|---|
| Commercial Rights |  |
| Program | Indication | Target |
Progress/Phase
|
Anticipated Milestones | Commercial Rights |
|---|---|---|---|---|---|
| CNS Diseases | |||||
|
CMP-002 |
SYNGAP1-related disorder | SYNGAP1 |
In between Phase 1 / 2 |
Clinical initiation in Q4 2026. Topline data 1H 2028. |
|
|
SHANK3 |
Phelan-McDermid Syndrome | SHANK3 |
in discovery & preclinical phase |
Development Candidate in 2027. |
|
|
CNS Program 1 |
CNS haploinsufficiency |
in discovery & preclinical phase |
|
||
|
CNS Program 2 |
CNS haploinsufficiency |
in discovery & preclinical phase |
|
||
| Metabolic Diseases | |||||
|
CMP-001 |
Urea Cycle Disorders | CPS1 |
in phase 1/2 Exploring potential partnership opportunities. |
|
|
| Collaborations | |||||
| Strategic research collaboration to identify and develop antisense oligonucleotide (ASO) drug candidates for multiple gene targets relevant to neurodegenerative and kidney disease indications. |
|
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Partnering to Unlock our Potential
Our RAP Platform® has broad therapeutic potential across any cell type underlying a disease driven by inadequate protein expression. Given the enormity of the opportunity, we can’t do it all alone. Partnering with biopharma companies with expertise in each therapeutic area is integral to maximizing the potential of our platform for patients. Our aim is to empower partners with a rapid, efficient approach to targeting the diseases that matter most to them.
We believe our technology and unparalleled understanding of regRNAs combined with the specificity and potency of ASOs can usher in a new era of genetic medicines.
