Onasemnogene Abeparvovec-xioi — Competitive Intelligence Brief

Onasemnogene Abeparvovec-xioi (Onasemnogene Abeparvovec-xioi) competitive landscape: 6 comparators, recent regulatory actions, upcoming PDUFA, patent timeline. Class: Gene therapy. Area: Neurology.

marketed Gene therapy SMN1 gene Neurology Biologic

Target snapshot

Onasemnogene Abeparvovec-xioi (Onasemnogene Abeparvovec-xioi) — Novartis Gene Therapies. Onasemnogene abeparvovec-xioi is a gene therapy that delivers a functional copy of the SMN1 gene via an adeno-associated virus vector to restore survival motor neuron protein production in patients with spinal muscular atrophy.

Comparator set (6 drugs)

Drug	Generic	Sponsor	Phase	Class	Target	Approval / PDUFA
Onasemnogene Abeparvovec-xioi TARGET	Onasemnogene Abeparvovec-xioi	Novartis Gene Therapies	marketed	Gene therapy	SMN1 gene
Onasemnogene Abeparvovec-brve	Onasemnogene Abeparvovec-brve	Novartis Pharmaceuticals	marketed	Gene Therapy	SMN1 gene
p53 gene therapy	p53 gene therapy	Shenzhen SiBiono GeneTech Co.,Ltd	marketed	Gene therapy	p53 (tumor suppressor protein)
rAd-p53 gene	rAd-p53 gene	Shenzhen SiBiono GeneTech Co.,Ltd	marketed	Gene therapy; oncolytic virus	p53 tumor suppressor gene
voretigene neparvovec-rzyl	voretigene neparvovec-rzyl	Spark Therapeutics, Inc.	marketed	Gene therapy	RPE65 gene
p53 gene with surgery	p53 gene with surgery	Shenzhen SiBiono GeneTech Co.,Ltd	marketed	Gene therapy (p53 tumor suppressor)	p53 (tumor protein p53)
p53 with chemotherapy	p53 with chemotherapy	Shenzhen SiBiono GeneTech Co.,Ltd	marketed	Gene therapy; recombinant adenovirus vector	p53 (tumor suppressor protein)

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 therapy class)

1. Spark Therapeutics, Inc. · 4 drugs in this class
2. MeiraGTx UK II Ltd · 3 drugs in this class
3. Novartis Gene Therapies · 2 drugs in this class
4. Kolon TissueGene, Inc. · 2 drugs in this class
5. Krystal Biotech, Inc. · 2 drugs in this class
6. Abeona Therapeutics, Inc · 2 drugs in this class
7. Larimar Therapeutics, Inc. · 1 drug in this class
8. 4D Molecular Therapeutics · 1 drug in this class
9. Medeor Therapeutics, Inc. · 1 drug in this class
10. MeiraGTx, LLC · 1 drug in this class