June 2023
Volume 64, Issue 8
Open Access
ARVO Annual Meeting Abstract  |   June 2023
Compassionate use of topical beremagene geperpavec (B-VEC) for the treatment of recurrent cicatrizing conjunctivitis in a patient with dystrophic epidermolysis bullosa
Author Affiliations & Notes
  • Alfonso L. Sabater
    University of Miami Health System Bascom Palmer Eye Institute, Miami, Florida, United States
  • Arianna Tovar
    University of Miami Health System Bascom Palmer Eye Institute, Miami, Florida, United States
  • Jennifer Gomez
    University of Miami Health System Bascom Palmer Eye Institute, Miami, Florida, United States
  • Trevor Parry
    Krystal Biotech, Pittsburgh, Pennsylvania, United States
  • Hubert Chen
    Krystal Biotech, Pittsburgh, Pennsylvania, United States
  • Brittani Agostini
    Krystal Biotech, Pittsburgh, Pennsylvania, United States
  • Suma Krishnan
    Krystal Biotech, Pittsburgh, Pennsylvania, United States
  • Footnotes
    Commercial Relationships   Alfonso Sabater Abbvie, Code C (Consultant/Contractor), GlaxoSmithKline, Code C (Consultant/Contractor), Brill Pharma, Code C (Consultant/Contractor), Laboratorios Sophia, Code C (Consultant/Contractor), TissueCor, Code O (Owner), LyoDrop, Code O (Owner), PlasmaCord, Code O (Owner); Arianna Tovar None; Jennifer Gomez None; Trevor Parry Krystal Biotech, Code E (Employment); Hubert Chen Krystal Biotech, Code E (Employment); Brittani Agostini Krystal Biotech, Code E (Employment); Suma Krishnan Krystal Biotech, Code E (Employment)
  • Footnotes
    Support  Beauty of Sight Foundation (Dr. Sabater), NIH Center Core Grant P30EY014801 (institutional), and Research to Prevent Blindness Unrestricted Grant GR004596 (institutional)
Investigative Ophthalmology & Visual Science June 2023, Vol.64, 787. doi:
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      Alfonso L. Sabater, Arianna Tovar, Jennifer Gomez, Trevor Parry, Hubert Chen, Brittani Agostini, Suma Krishnan; Compassionate use of topical beremagene geperpavec (B-VEC) for the treatment of recurrent cicatrizing conjunctivitis in a patient with dystrophic epidermolysis bullosa. Invest. Ophthalmol. Vis. Sci. 2023;64(8):787.

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      © ARVO (1962-2015); The Authors (2016-present)

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Abstract

Purpose : Dystrophic epidermolysis bullosa (DEB) is a rare genetic blistering skin disease caused by mutations in the COL7A1 gene. A subset of individuals with DEB may develop abrasions, blistering and scarring of the ocular surface that can lead to impaired vision. Current therapies are limited to removal of scar tissue and ophthalmic lubrication. Using a replication deficient herpes simplex virus type 1 (HSV-1)-based gene delivery platform, topical beremagene geperpavec (B-VEC) has been engineered to deliver functional human type VII collagen (COL7). B-VEC has been evaluated in the skin of patients with DEB and in immunocompetent BALB/c mice corneas. To our knowledge, our patient is the first human treated with a topical ocular gene therapy.

Methods : Compassionate use study in a 13-year-old male with DEB at the Bascom Palmer Eye Institute (Miami, FL) who presented with bilateral advanced cicatrizing conjunctivitis. He had a history of repeated symblepharon lysis surgeries with posterior recurrence, and bilateral limbal stem cell deficiency. Surgical symblepharon lysis of his right eye with subsequent weekly topical applications of B-VEC (5×109 PFU/mL) were performed until corneal epithelium was healed, followed by monthly topical applications.

Results : Prior to the surgery, he had severe ankyloblepharon that reached the central cornea in both eyes. His visual acuity (VA) was hand motion (HM) in the right eye and 20/400 in left eye. Immediately after surgery, an amniotic membrane and a bandage contact lens (BCL) were placed. After removal of the BCL 1 month later, a corneal epithelial defect and peripheral neovascularization were noted. Four months after the surgery the symblepharon had not recurred and the cornea was completely re-epithelized. He went from HM VA to 20/100 after surgical intervention and B-VEC treatment and is currently being scheduled to undergo the same procedures in his left eye.

Conclusions : Topical ocular application of B-VEC in this 13-year-old patient with DEB was well-tolerated with no signs of ocular HSV-1-like disease. VA improved without recurrence of symblepharon. Based on this first case, B-VEC has the potential to be a safe and effective treatment for recurrent cicatrizing conjunctivitis in patients with DEB. Longer follow-up and future, larger studies are required for further and consistent evidence.

This abstract was presented at the 2023 ARVO Annual Meeting, held in New Orleans, LA, April 23-27, 2023.

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