Announced details
MDL-202 demonstrated exceptionally strong DMPK gene suppression following systemic administration to mice. MDL-103 markedly suppressed DUX4 target genes at a low dose in an FSHD disease model. Specific figures are Not disclosed.
Affinia Therapeutics is a collaborative research partner. The relationship with the University of Massachusetts is Not disclosed except for the use of a disease model developed by the university.
Affinia Therapeutics is a collaborative research partner that developed the muscle-tropic capsid ATC-187. The University of Massachusetts is the university that developed a xenograft disease-model mouse implanted with muscle cells derived from FSHD patients. Other information, including location, representative, capital, and year established, is Not disclosed.
Boston, United States (ASGCT venue)
In this announcement the company addresses none of Contract Value / Offering price / Impact on Business Performance.
29th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT)
| Item | Details |
|---|---|
| Names of Products / Services Covered | MDL-202, MDL-103, CRISPR-GNDM® |
| Counterparty | Affinia Therapeutics, University of Massachusetts |
| Counterparty overview | Affinia Therapeutics is a collaborative research partner that developed the muscle-tropic capsid ATC-187. The University of Massachusetts is the university that developed a xenograft disease-model mouse implanted with muscle cells derived from FSHD patients. Other information, including location, representative, capital, and year established, is Not disclosed. |
| Relationship with the Company | Affinia Therapeutics is a collaborative research partner. The relationship with the University of Massachusetts is Not disclosed except for the use of a disease model developed by the university. |
| Launch date | 11 to 15 May 2026 (scheduled presentation at ASGCT) |
| Applicable area | Treatment of myotonic dystrophy type 1 (DM1), Treatment of facioscapulohumeral muscular dystrophy (FSHD) |
| Location | Boston, United States (ASGCT venue) |
| Quantitative Effects | MDL-202 demonstrated exceptionally strong DMPK gene suppression following systemic administration to mice. MDL-103 markedly suppressed DUX4 target genes at a low dose in an FSHD disease model. Specific figures are Not disclosed. |
| Source | 29th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) |
Items this document does not state (9)
These are items the company does not state in this document. We do not leave them blank, nor fill them in from other sources or by inference.
The reasons the company gives
The company's own explanation, not something inferred from the numbers.The proprietary CRISPR-GNDM® technology suppresses expression of disease-causing genes in a muscle-selective and sustained manner without cutting double-stranded DNA.