Details of the Drug-Related molecule(s) Interaction Atlas

General Information of Drug (ID: DMDY7RR)

• Drug Name: BIIB112 Drug Info
• Synonyms: cotoretigene toliparvovec

Indication

Disease Entry	ICD 11	Status	REF
X-linked retinitis pigmentosa	9B70	Phase 3	[1]

• Cross-matching ID: TTD Drug ID: DMDY7RR

Molecule-Related Drug Atlas

Drug(s) Targeting X-linked retinitis pigmentosa GTPase regulator (RPGR)

Drug Name	Drug ID	Indication	ICD 11	Highest Status	REF
BIIB-088	DMUSJ3D	Retinitis pigmentosa	9B70	Phase 2/3	[3]
AGTC-501	DMTS51C	Retinitis pigmentosa	9B70	Phase 1/2	[4]
AAV-RPGR	DM7TF49	Retinitis pigmentosa	9B70	Phase 1/2	[5]

Molecular Interaction Atlas of This Drug

Drug Therapeutic Target (DTT)

DTT Name	DTT ID	UniProt ID	MOA	REF
X-linked retinitis pigmentosa GTPase regulator (RPGR)	TTHBDA9	RPGR_HUMAN	Not Available	[2]

References

• [1] ClinicalTrials.gov (NCT03584165) A Long-term Follow-up Study to Evaluate the Safety and Efficacy of Retinal Gene Therapy in Subjects With Choroideremia Previously Treated With Adeno-Associated Viral Vector Encoding Rab Escort Protein-1 (AAV2-REP1) and in Subjects With X-Linked Retinitis Pigmentosa Previously Treated With Adeno-Associated Viral Vector Encoding RPGR (AAV8-RPGR) in an Antecedent Study. U.S.National Institutes of Health.
• [2] Changes in Retinal Sensitivity Associated With Cotoretigene Toliparvovec in X-Linked Retinitis Pigmentosa With RPGR Gene Variations. JAMA Ophthalmol. 2023 Mar 1;141(3):275-283.
• [3] ClinicalTrials.gov (NCT04850118) A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With X-linked Retinitis Pigmentosa Caused by RPGR Mutations. U.S. National Institutes of Health.
• [4] ClinicalTrials.gov (NCT03316560) Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations. U.S. National Institutes of Health.
• [5] Clinical pipeline report, company report or official report of MeiraGTx.