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CTI-1601 — Competitive Intelligence Brief

CTI-1601 (CTI-1601) competitive landscape: 6 comparators, recent regulatory actions, upcoming PDUFA, patent timeline. Class: Gene therapy. Area: Neurology.

phase 2 Gene therapy SOD1 gene Neurology Biologic Live · refreshed every 30 min

Target snapshot

CTI-1601 (CTI-1601) — Larimar Therapeutics, Inc.. CTI-1601 is a gene therapy that aims to restore motor neuron function in patients with amyotrophic lateral sclerosis (ALS).

Comparator set (6 drugs)

Auto-detected by same drug class / molecular target / therapeutic area, ranked by phase + recency. Override with ?vs=slug1,slug2,….

DrugGenericSponsorPhaseClassTargetApproval / PDUFA
CTI-1601 TARGET CTI-1601 Larimar Therapeutics, Inc. phase 2 Gene therapy SOD1 gene
Tivanisiran sodium ophthalmic solution Tivanisiran sodium ophthalmic solution Sylentis, S.A. phase 3 antisense oligonucleotide SOD1 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
rAd-p53 gene rAd-p53 gene Shenzhen SiBiono GeneTech Co.,Ltd marketed Gene therapy; oncolytic virus p53 tumor suppressor gene
p53 with chemotherapy p53 with chemotherapy Shenzhen SiBiono GeneTech Co.,Ltd marketed Gene therapy; recombinant adenovirus vector p53 (tumor suppressor protein)
p53 gene therapy p53 gene therapy Shenzhen SiBiono GeneTech Co.,Ltd marketed Gene therapy 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

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Cite this brief

Drug Landscape (2026). CTI-1601 — Competitive Intelligence Brief. https://druglandscape.com/ci/cti-1601. Accessed 2026-05-14.

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