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RTW’s Rocket takes off on good clinical data

RTW’s Rocket takes off on good clinical data – RTW Venture Fund notes positive data updates on four clinical trials of Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT). This is the largest holding in RTW’s portfolio, representing about 30% of NAV.

Rocket is advancing an integrated and sustainable pipeline of first-in-class genetic therapies that correct the root cause of complex and rare childhood disorders. Between 6th and 8th of December, the portfolio company shared four positive data readouts, two of which were updates to previously reported data and two of which represented first-time Phase I readouts for:

  • Update to its lentiviral vector (LVV)-based gene therapy programs for the treatment of Fanconi Anaemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer
  • Update to its Leukocyte Adhesion Deficiency-I (LAD-I) program, a severe paediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal
  • First-time readout for Pyruvate Kinase Deficiency (PKD) a rare, monogenic red blood cell disorder
  • First-time readout for an adeno-associated virus (AAV)-based gene therapy for Danon disease, a devastating, paediatric heart failure condition.

Given highly anticipated positive news on clinical trials progress Rocket’s share price traded up +83.4% on 09 December 2020, translating into a materially uplift in RTW’s NAV.

 

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