CT001 — Competitive Intelligence Brief

CT001 (CT001) competitive landscape: 2 comparators, recent regulatory actions, upcoming PDUFA, patent timeline. Class: Gene editing therapy. Area: Neurology.

phase 2 Gene editing therapy SMN1 gene Neurology Small molecule

Target snapshot

CT001 (CT001) — Cessatech A/S. CT001 is a gene editing therapy that uses CRISPR-Cas9 to edit the SMN1 gene.

Comparator set (2 drugs)

Drug	Generic	Sponsor	Phase	Class	Target	Approval / PDUFA
CT001 TARGET	CT001	Cessatech A/S	phase 2	Gene editing therapy	SMN1 gene
Onasemnogene Abeparvovec-brve	Onasemnogene Abeparvovec-brve	Novartis Pharmaceuticals	marketed	Gene Therapy	SMN1 gene
Onasemnogene Abeparvovec-xioi	Onasemnogene Abeparvovec-xioi	Novartis Gene Therapies	marketed	Gene therapy	SMN1 gene

Recent regulatory actions (last 90 days)

No regulatory actions in the last 90 days for this set.

Upcoming PDUFA dates (next 180 days)

No PDUFA dates in the next 180 days for this set.

Patent timeline (forward window)

No upcoming patent expiries for this set.

Sponsor landscape (Gene editing therapy class)

1. Cessatech A/S · 1 drug in this class