dm+d

Unassigned

New Medicines

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

Information

New molecular entity
Holostem Terapie Avanzate
Holostem Terapie Avanzate

Development and Regulatory status

None
Phase III Clinical Trials
None
Yes

Category

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
Implantation