Degrading SPTLC1 RNA to Reduce Lipid Production in ALS with LTX-002 ASO
Time: 2:00 pm
day: Conference Day Two
Details:
- Translating ASO potency in mice to CNS target engagement with repeat IT dosing target engagement in rodents with repeat IT dosing in patients
- Reviewing NfL reduction as a surrogate endpoint at 3 months for 12 months sustained reduction
- Establishing SPTLC1 ASO LTX-002 for safety, target engagement and biodistribution to critical CNS regions in NHP brains
