New Medicines

LAMB-3 dependent junctional epidermolysis bullosa (JEB) in patients aged 6 months to 55 years


New molecular entity
Holostem Terapie Avanzate
Holostem Terapie Avanzate

Development and Regulatory status

Phase III Clinical Trials
Mar 15Granted orphan drug status in EU [2].


Ex-vivo-expanded autologous human keratinocytes containing epidermal stem cells transduced with a LAMB3-encoding retroviral vector, administered as a cutaneous implant
JEB is a rare inherited skin disorder in which skin is fragile and minor everyday knocks and friction can lead to blisters and raw areas. It is an autosomal recessive condition., and can be lethal early in life. Prevalence at birth ranges from 1 in 110,000 to 455,000 worldwide, with estimates varying between countries [1]. In 2015, the EMA indicated that EB affected approximately 31,000 people in the EU [2].
LAMB-3 dependent junctional epidermolysis bullosa (JEB) in patients aged 6 months to 55 years

Trial or other data

Oct 21Pivotal PII/III HOLOGENE 5 single-arm study to assess the efficacy and safety of autologous fibrin-cultured epidermal grafts containing epidermal stem cells genetically modified for restoration of epidermis in patients with JEB starts (EudraCT 2018-000261-36). The main objective of the trial is to assess at 12 months follow-up as percentage of success based on the following assessments: clinical performance as % of re-epithelization in the absence of blisters measured by Investigator through a software named Holosnapp; functional evaluation based on laboratory analyses and mechanical assessment; and patient outcome assessment based on patient improvement perception on the transplanted areas. Six male and female patients between 6-month-old and 55-year-old will be recruited in two European countries (not stated) [3].
Sep 21Rationale and design of PII/III HOLOGENE 5 trial published in Frontiers in Genetics journal [4].