Basic Information

Gene symbol E2F1 Synonyms E2F-1, RBAP1, RBBP3, RBP3 Type of gene protein-coding
Gene perturbation-related omics dataset PerturbAtlas | PertOrg
Description E2F transcription factor 1

Gene symbol SPAM1 Synonyms HEL-S-96n, HYA1, HYAL1, HYAL3, HYAL5, PH-20, PH20, SPAG15 Type of gene protein-coding
Description sperm adhesion molecule 1

GTO ID GTC3984
Trial ID NCT03284268
Disease Retinoblastoma
Altered gene E2F1|PH20
Therapeutic/Target gene Therapeutic gene
TherapyDNA oncolytic virus
Treatment VCN-01
PhaseNot Applicable
Recruitment statusCompleted
TitlePhase I Study, Single Site, Open Label With Dose Escalation, for Evaluate Safety and the Oncolitic Adenovirus VCN-01 Activity in Patients With Refractory Retinoblastoma
Year2017
CountrySpain
Company sponsorFundació Sant Joan de Déu
Other ID(s)FSJD-RTB-2015
Vector information
Vectoradenovirus
ConstructAd5-E2F-delta24-RGD -PH20
Vector typeoncolytic adenovirus (Ad5 based)
Transgene/Inserted geneHyaluronidase (PH20), which enables the virus to degrade the extracellular matrix, disrupt the tumor stroma, thus enhancing the intratumoral spreading of the virus.
Viral genome modificationVCN-01 incorporates an RGKD integrin motif in the fiber shaft, an effective method for liver detargeting and tumor retargeting.
Additional featureHyaluronidase (PH20)

Clinical Result

Cohort1: dose level 1
Administration route intravitreal injection
Dosage 2E9 viral particules/eye
Age Child
Cohort2: dose level 2
Administration route intravitreal injection
Dosage 2E10 viral particules/eye
Age Child
Cohort3: dose level 3
Administration route intravitreal injection
Dosage 2E11 viral particules/eye
Age Child

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