Biotech Disruption - Gene Editing
CRISPR Therapeutics on the Move
Gene editing suggests game changing therapeutic options. It is set to improve the outlook dramatically for patients affected by diseases with no effective therapy offerings for the time being. Gene editing holds promise of transforming the treatment of diseases like cancer and rare diseases.
The Swiss biotech company CRISPR Therapeutics with partner Vertex Pharmaceuticals has filed Exa-Cel for approval in the US, EU and UK and may get approval this year. If approved it will become the first CRISPR-Cas9 gene editing therapeutic on the market. Exa-Cel has shown strong efficacy in the treatment of the devastating blood diseases sickle cell disease (SCD) and transfusion-dependent beta thalassemia. SCD is a genetic disorder that causes strokes, organ damage, severe pain and early death affecting around 100,000 patients in the US. Furthermore
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Exa-Cel's clinical efficacy and cost of therapy effectiveness (single once for all treatment) justifies a high price. A reference drug using another technology and recently approved is bluebird bio's gene therapy lovo-cel for the treatment of beta thalassemia priced at USD 2.8m. Accordingly the US pricing of Exa-Cel will probably be USD 2m+.
CRISPR Therapeutics excels with a strong technology platform including - in addition to gene editing - allogenic and autologous cell therapy and a diversified pipeline within blood diseases, oncology and diabetes.
VRAA BIOTECH, currently including 14 positions, combines holdings of mature tier 1 and 2 biotechs with high potential emergent biotechs. CRISPR Therapeutics was included in the VRAA BIOTECH portfolio in April this year and has surged 56% since then. The VRAA BIOTECH top performing positions: TG Therapeutics continues its stellar performance up 195% this year, Immunogen (+189%) and Omeros (+144%). The VRAA BIOTECH portfolio is up 30% year to date. That compares well to the Black Rock iShares Biotechnology ETF benchmark, up with modest 1%. Active portfolio management is worth the effort; at least in biotech.