Biotech Disruption - Gene Editing

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

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.

To view or add a comment, sign in

More articles by Lars Hatholt

  • Strong H1 for VRAA BIOTECH (+28%): Outperforming with 23% Avg. ROI

    Strong H1 for VRAA BIOTECH (+28%): Outperforming with 23% Avg. ROI

    VRAA BIOTECH achieved a 28% return in H1 2024, outperforming the Nasdaq Biotech index. Since 2015, VRAA BIOTECH has…

  • VRAA Biotech: Outsized Returns in the Biotech Sector

    VRAA Biotech: Outsized Returns in the Biotech Sector

    The VRAA Biotech portfolio was up 40% in 2023 and has consistently outperformed benchmark biotech ETFs, achieving an…

  • VRAA BIOTECH: Strong Performance once again

    VRAA BIOTECH: Strong Performance once again

    The VRAA BIOTECH portfolio is heading for a strong 2023 performance, up 31% until now. The biotech bear market is in…

  • VRAA BIOTECH: Up 35% In Q2

    VRAA BIOTECH: Up 35% In Q2

    The VRAA BIOTECH PM strategy combines mature biotech stocks (tier-1) with less mature biotechs (tier 2-4). The…

  • Navigating the Biotech Deadlock

    Navigating the Biotech Deadlock

    In Q1 the VRAA BIOTECH portfolio (+3%) outperformed the Nasdaq Biotech ETF IBB (-2%) while the S&P Biotech XBI ETF…

  • VRAA BIOTECH UP 18% IN 2022

    VRAA BIOTECH UP 18% IN 2022

    The VRAA BIOTECH portfolio gained 18% in 2022 in a challenging stock market thanks to a 19% surge in H2. The VRAA…

  • Brutal Biotech

    Brutal Biotech

    The stock markets are bleeding, and that goes especially for growth stocks like biotech amid rising interest rates…

  • 2021 One Of The Worst Years In Biotech

    2021 One Of The Worst Years In Biotech

    It may sound strange with 2021 being one of the worst yeas for biotech stocks in a year of surging stock markets. Never…

    2 Comments
  • US NASDAQ Biotech Stocks hit hard by Covid-19 Vaccines

    US NASDAQ Biotech Stocks hit hard by Covid-19 Vaccines

    US NASDAQ biotech stocks seem to have performed quite well this year with the US NASDAQ biotech index up by 15%. Below…

Insights from the community

Others also viewed

Explore topics