[signal.] — portfolio & asset diligence

Asset	Indication	Target	Stage	Outcome	Posture	Evidence
Anti-Amyloid Monoclonal (Early AD) Alzheimer disease	Alzheimer disease	APPMAPT	Phase 2	Clinical entry	Mixed· medium conf	4
CFTR Corrector Triple Combination Cystic fibrosis	Cystic fibrosis	CFTR	Phase 3	Licensed	Supportive· high conf	3
FXN Frataxin Restoration Program Friedreich ataxia	Friedreich ataxia	FXN	IND-enabling	Active	Mixed· low conf	2
GBA1 Gene Therapy for Parkinson Disease Parkinson disease	Parkinson disease	GBA1	Preclinical	Dormant	Concerning· low conf	2
HTT-Lowering ASO for Huntington Disease Huntington disease	Huntington disease	HTT	Phase 1	Active	Concerning· medium conf	3
JAG1 Modulator for Alagille Syndrome Alagille syndrome	Alagille syndrome	JAG1NOTCH2	Preclinical	Active	Mixed· medium conf	4
MECP2 Dosage-Controlled Gene Therapy Rett syndrome	Rett syndrome	MECP2	Discovery	Active	Insufficient· low conf	2
Microdystrophin Gene Transfer (DMD) Duchenne muscular dystrophy	Duchenne muscular dystrophy	DMD	Phase 2	Clinical entry	Mixed· medium conf	3
SCN1A Upregulation for Dravet Syndrome Dravet syndrome	Dravet syndrome	SCN1A	Phase 1/2	Clinical entry	Supportive· high conf	3
SMN2 Splicing Enhancer (Oral) Spinal muscular atrophy	Spinal muscular atrophy	SMN1SMN2	Phase 1	Clinical entry	Mixed· high conf	3
SOD1 Antisense Oligonucleotide Amyotrophic lateral sclerosis	Amyotrophic lateral sclerosis	SOD1	Phase 1/2	Clinical entry	Supportive· medium conf	3
SOD1 Stabilizer (Small Molecule) Amyotrophic lateral sclerosis	Amyotrophic lateral sclerosis	SOD1	Preclinical	Active	Mixed· low conf	3