Pre­vail's gene ther­a­py scores or­phan, rare pe­di­atric dis­ease priv­i­leges; Ger­many's ITM se­cures €40M loan

Pre­vail Ther­a­peu­tics’ gene ther­a­py for Gauch­er dis­ease now has both or­phan drug and rare pe­di­atric dis­ease des­ig­na­tions from the FDA. The lat­ter was specif­i­cal­ly for the treat­ment of neu­rono­path­ic Gauch­er dis­ease, the most se­vere form of the con­di­tion. PR001 is de­signed to trans­fer the GBA1 gene to pa­tients.

→ Pri­vate­ly-held biotech ITM (Iso­topen Tech­nolo­gien München) has scored a €40 mil­lion loan from the Eu­ro­pean In­vest­ment Bank to fund the de­vel­op­ment of di­ag­nos­tics and ther­a­pies to ad­dress a range of can­cers such as neu­roen­docrine tu­mors and bone metas­tases.

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