Platform

Science

AAV gene therapy platform and lead programs

Our Platform

AAV Gene Therapy

Arcivus combines computational biology with precision AAV vector engineering to develop gene therapies for rare neurological disorders. Our platform integrates target validation, vector optimization, and translational science into a unified pipeline.

Beginning with DYT-SGCE myoclonus-dystonia, we're building scalable infrastructure for gene replacement therapy across multiple rare disease indications.

Focus Areas

Therapeutic Areas

Neurological

Active

Movement disorders and rare neurological conditions amenable to AAV-mediated gene replacement.

Muscular

Discovery

Genetic muscle diseases where dystrophin-family proteins present therapeutic targets.

Metabolic

Discovery

Inborn errors of metabolism with central nervous system involvement and single-gene etiology.

Lead Program: DYT-SGCE

Genetic identity, variant analysis, and therapeutic strategy

Genetic Identity

Primary Variant: SGCE (Pathogenic)

Likely Pathogenic Variant (LPV)

Transcript: NM_003919.3
DNA change: c.108dup
Protein effect: p.Val37SerfsTer32
Mechanism: 1 bp duplication in exon 3 → reading frame shift → NMD → complete protein loss
Inheritance: Autosomal dominant, maternally imprinted (paternal allele affected)

Secondary Variant: THAP1 (VUS)

Variant of Uncertain Significance

Transcript: NM_018105.3
DNA change: c.496G>A
Protein effect: p.Ala166Thr

Therapeutic Strategy

Primary Strategy

AAV Gene Replacement

Mutation-agnostic. Delivers full-length functional protein via episomal transgene. Strong CNS clinical precedent.

Secondary Strategy

Maternal Allele De-repression

Reactivate the silenced wildtype maternal allele via targeted epigenetic editing (dCas9-TET1 or ASO).

Long-term Watch

Prime Editing

The only editing tool capable of deleting a 1 bp insertion. Could correct c.108dup at the genomic level.

Transient / Backup

mRNA (LNP)

Repeat-dose mRNA delivery. No genome modification. Limited by CNS penetration and dosing frequency.

Comparative Analysis

Criterion	AAV Replacement	Epigenetic Reactivation	Prime Editing	mRNA (LNP)
Implementation complexity	Low	Medium	Very High	Very High
CNS clinical validation	Strong	Preclinical	Preclinical	Early
Genome modification	None	Epigenome only	1 nt deletion	None
Durability (single dose)	Lifelong*	Uncertain	Lifelong*	Transient
Vector packaging	Single AAV	N/A	Dual AAV	LNP
Off-target risk	Minimal	Moderate	Moderate	Low
Compatible with c.108dup	Yes	Yes	Yes	Yes

* In post-mitotic neurons. AAV episomal DNA persists without cell division.