Evidence map›Paper›PMID 40667654›Full record

ArticleThe Journal of dermatology2025

Efficacy and Safety of the Topical Gene Therapy Beremagene Geperpavec-svdt (B-VEC) in an Open-Label Study of Japanese Subjects With Dystrophic Epidermolysis Bullosa.

Ken Natsuga, Daisuke Tsuruta, Shota Takashima, Chiharu Tateishi, Masaaki Takatoku, Brittani Agostini, Sarrah Mailliard, Nicholas J Reitze, Rebecca T Beacham, Alexia M Cardiges and 3 more

Abstract readMulticenter Study
PubMed Full text at PMC Publisher
In one paragraph

Article in The Journal of dermatology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

3 citing papers in PubMed.

  1. Review
  2. Review
  3. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

13 authors.

Ken NatsugaDepartment of Dermatology, Graduate School of Medicine, Hokkaido University, Sapporo, Japan.ORCID https://orcid.org/0000-0003-3865-6366
Daisuke TsurutaDepartment of Dermatology, Graduate School of Medicine, Osaka Metropolitan University, Osaka, Japan.
Shota TakashimaDepartment of Dermatology, Graduate School of Medicine, Hokkaido University, Sapporo, Japan.
Chiharu TateishiDepartment of Dermatology, Graduate School of Medicine, Osaka Metropolitan University, Osaka, Japan.
Masaaki TakatokuKrystal Biotech, Inc., Pittsburgh, Pennsylvania, USA.
Brittani AgostiniKrystal Biotech, Inc., Pittsburgh, Pennsylvania, USA.
Sarrah MailliardKrystal Biotech, Inc., Pittsburgh, Pennsylvania, USA.
Nicholas J ReitzeKrystal Biotech, Inc., Pittsburgh, Pennsylvania, USA.
Rebecca T BeachamKrystal Biotech, Inc., Pittsburgh, Pennsylvania, USA.
Alexia M CardigesKrystal Biotech, Inc., Pittsburgh, Pennsylvania, USA.
Michael J JohnstonKrystal Biotech, Inc., Pittsburgh, Pennsylvania, USA.
Ramakrishna EdukullaKrystal Biotech, Inc., Pittsburgh, Pennsylvania, USA.
Suma M KrishnanKrystal Biotech, Inc., Pittsburgh, Pennsylvania, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Dystrophic epidermolysis bullosa (DEB) patients have pathogenic variants in COL7A1, leading to skin fragility, blistering, and scarring. Beremagene geperpavec-svdt (B-VEC) is a replication-defective herpes simplex virus type 1 (HSV-1)-based gene therapy vector administered topically to deliver functional COL7A1 to DEB wounds. In a United States (US) Phase 3 study, B-VEC significantly improved wound healing at 3 and 6 months compared to placebo, and in a US open-label extension (OLE) study, weekly B-VEC administration was well tolerated for up to 112 weeks. The present study was conducted to confirm the efficacy and safety of B-VEC in a cohort of Japanese DEB patients receiving weekly B-VEC treatment (4.0 × 10

Indexed as

Collagen Type VIIEpidermolysis Bullosa DystrophicaGenetic TherapyGenetic VectorsAdministration, TopicalAdolescentAdultAgedChildEast Asian PeopleFemaleHerpesvirus 1, HumanHumansJapanMaleMiddle AgedCOL7A1 protein, humanCollagen Type VIIB‐VECclinical trialdystrophic epidermolysis bullosaJapantopical therapy

Identifiers

PMID40667654
PMCPMC12530466

What Socratic holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.