Filsuvez has the potential to become the first treatment approved for epidermolysis bullosa, a rare genetic condition where skin can tear and blister at even the slightest touch.

Public Company Edition: Moderna leveraged its all-time high stock price to raise $1.34bn on the same day it reported data from a Phase I trial of its COVID-19 vaccine. Several other companies also took advantage of biopharma’s rising value.

Zolgensma shows that a gene therapy can be a commercial hit – but there is still plenty to be wary of in this cutting-edge field.

Public Company Edition: Jefferies report notes robust IPO market, but SVB Leerink predicts a financing slowdown. Also, Mirati raises cash for KRAS programs after Amgen's ASCO data and a strategic shift results in job cuts at ImmunoGen.