Syndeo Life, headquartered in Baltimore, Maryland, is a biotechnology company and Johns Hopkins University spinout developing gene therapies and biomarkers for neurodegenerative diseases driven by TDP-43 dysfunction. Its lead program, AAV9-CTR, is designed to restore TDP-43 splicing repression across the transcriptome in amyotrophic lateral sclerosis (ALS). Syndeo is also advancing a proprietary fluid biomarker platform designed to support patient selection and target engagement, with potential expansion into frontotemporal dementia (FTD) and Alzheimer’s disease.
Solutions
Syndeo Life is developing AAV9-CTR, a one-time intrathecal gene therapy for diseases associated with TDP-43 dysfunction. AAV9-CTR delivers CTR, a chimeric TDP-43/RAVER1 protein designed to restore TDP-43 splicing repression across the transcriptome. The therapy also includes an autoregulatory element designed to limit overexpression.
Unlike approaches that target a single downstream cryptic exon, AAV9-CTR addresses the broader effects of TDP-43 loss of function. Syndeo is pairing its gene therapy with its proprietary cryptic HDGFL2 fluid biomarker, which is detectable in blood and cerebrospinal fluid (CSF) and is being developed to support patient selection and measurement of target engagement.
Because TDP-43 pathology occurs in more than 97% of cases, the company is initially developing AAV9-CTR for ALS, with potential expansion into FTD and Alzheimer’s disease.
Strengths
- Transcriptome-Wide Rescue: AAV9-CTR is designed to restore TDP-43 splicing repression across the transcriptome, addressing hundreds of cryptic exon errors rather than a single downstream target.
- Mechanistic Biomarker: Syndeo’s proprietary cryptic HDGFL2 fluid biomarker is detectable in blood and CSF and is being developed to support early patient identification, target engagement and treatment monitoring.
- Preclinical Validation: In a rodent ALS model, AAV-CTR administered after symptom onset improved motor performance and body weight, preserved motor neurons and rescued survival compared with controls.
- Protected Platform: Syndeo has an exclusive option to its therapeutic IP and has exclusively licensed its biomarker IP from Johns Hopkins.

Meet the Team
From left to right:
Jonathan Ling, Ph.D. | Scientific Founder & CTO
Li Chin Wong, Ph.D. | Co-Founder & CSO
Philip Wong, Ph.D. | Scientific Founder