February 6, 2024 5:06pm

With Monday’s advance/Decline (A/D) line was 11/23 and todays was 31/4

Pre-open indication: 1 Hit and 3 Miss

News: Ultragenyx Pharmaceutical (RARE +1.13) announced data demonstrating treatment with UX111 (ABO-102) AAV gene therapy resulted in rapid and sustained decreased levels of heparan sulfate (HS) in cerebrospinal fluid (CSF) in patients with Sanfilippo syndrome type A (MPS IIIA), and that sustained reduction in CSF HS exposure over time was correlated with improved long-term cognitive development. Study patients treated with UX111 have maintained their communication skills despite being the age in which regression begins to occur,

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A slight sigh of relief, I qualify the sector’s equity’s movement; with an emphasis on which company is gaining or losing stride in “our” portfolio in the short-term

It’s not hard to be right so often, it’s about refining expectation, defining insight and NOT being indentured to ANY financial institution!


Tuesday: The Dow closed UP +141.24 points or +0.37%, the S&P closed UP +11.42 points +0.23% while the Nasdaq closed DOWN +11.32 points or +0.07%



We need to more than consider the economic environment - rising rates and inflation to comprehend the micro re “our” universe of cell and gene therapy companies …

Indexes rose on Tuesday as the latest batch of corporate earnings and the timeline for rates cuts from the Fed were being assessed.


Tuesday’s RegMed Investors’ (RMi) pre-open: “sector’s investors are conflicted as share pricing declines contaminate potential buy attitude. Another weak aftermarket forecasting a drag on the sector.” https://www.regmedinvestors.com/articles/13316


Pre-open Indications: 1 Hit < Prime Medicine (PRME +$0.32) > 3 Miss < Sage Therapeutics (SAGE +$0.45), Vericel (VCEL +$0.94), Ionis Pharmaceuticals (IONS -$0.31)>


Advance/Decline (A/D) Line:

  • Tuesday’s advance/line lines at the open was positive with 28 incliners, 5 decliners and 2; ending with a negative close of 31 incliners, 4 decliners and 0 flat


Ebb and flow of MY covered sector cell and gene therapy session daily “endings”:

Q1/24: February, 2 negative and 2 positive closes

  • January: 2 holiday, 12 negative and 8 positive closes


Key Metrics:

The iShares NASDAQ Biotechnology (IBB) and the SPDR S&P ETF (XBI) indicators:

  • Tuesday, the IBB was up +0.96% and the XBI was up +1.53%

The CBOE Volatility Index (VVIX: INDEX) tracked:

  • Tuesday was down -0.65 point or -4.75% at 13.02


Tuesday’s Closing Down (4 of 4):

  • Fate Therapeutics (FATE -$0.62 after Monday’s -$0.16),
  • Ionis Pharmaceuticals (IONS -$ after Monday’s -$0.32),
  • Sangamo Therapeutics (SGMO -$0.0393 after Monday’s +$0.0684),
  • Bellicum Pharmaceuticals (BLCM -$0.0028 after Monday’s +$0.0104),
  • Brainstorm Cell therapeutics (BLCM -$),

Tuesday’s Closing Up (10 of 31):

  • CRISPR Therapeutics (CRSP +$2.37 after Monday’s -$0.24
  • Beam Therapeutics (BEAM +$1.95 after Monday’s +$0.01
  • Intellia Therapeutics (NTLA +$1.73 after Monday’s -$0.45
  • Alnylam Pharmaceuticals (ALNY +$1.48 after Monday’s -$2.36),
  • Ultragenyx Pharmaceuticals (RARE +$1.13 after Monday’s +$1.74)
  • Vericel (VCEL +$0.94 after Monday’s +$0.33)
  • Regenxbio (RGNX +$0.73 after Monday’s +$0.08)
  • Solid Biosciences (SLDB +$0.55 after Monday’s -$0.25),
  • Verve Therapeutics (VERV +$0.65 after Monday’s -$0.20
  • Adverum Biotechnologies (ADVM +$0.59 after Monday’s +$0.49)


Q1/24 – February

Tuesday closed positive with 31 incliners, 4 decliners and 0 flat

(2/5) Monday closed negative with 12 incliners, 23 decliners and 0 flat


The BOTTOM LINE: From the title, once the cork is out of the bottle, the fizz is dissipated and drunk …

And the bottle is barreled, wait for share pricing to begin rolling downward with a clang!


Think of this quote, "Now traders are wondering if instead of whether we'll get a soft landing or recession, whether we could have no landing or re-acceleration this year," said Matthew Weller, global head of research at FOREX.com. <Reuters>


What more can I write; I have said it all – read the weeklies and know I have been RIGHT!

  • Adding pressure was U.S. Treasuries, with 10-year yields up for second day straight and hitting their highest level since late January.

Geo-political risk coming at you: The Biden administration has limited its previous retaliation to over 165 attacks by Iran-supported militias on U.S. forces and a drone attack killed three U.S. troops in Jordan.

Reiterating … again, “the cell and gene therapy sector continues to “recalibrate” its share pricing expectations and I don't think this … adjustment is completely over.”

  • Earnings and guidance are beginning 2/5 as Alnylam Pharmaceuticals (ALNY) and Sage Therapeutics (SAGE) along with Blueprint Medicine (BPMC) on 2/14 announcing Q4 and FY23 which WILL be crucial to continue downside force in the sector as history has taught us i.e., investors.

Today’s business news affecting “our” universe:

  • Benchmark 10-year notes fell to 4.09%, after reaching an 11-day high of 4.177% on Monday.
  • The dollar index, which tracks the greenback against a basket of currencies of other major trading partners, was down 0.3% at 104.17.

The top three (3) performing in the session:

  • Tuesday: CRISPR Therapeutics (CRSP), Beam therapeutics (BEAM) and Intellia Therapeutics (NTLA)
  • Monday: Ultragenyx Pharmaceuticals (RARE), Adverum Biotechnologies (ADVM) and Vericel (VCEL)

The worst three (3) in the session:  

  • Tuesday: Fate Therapeutics (FATE), Ionis Pharmaceuticals (IONS) and Sangamo Therapeutics (SAGE)
  • Monday: Blueprint Medicine (BLMC), Alnylam Pharmaceuticals (ALNY) and Sage Therapeutics (SAGE)

Welcome to my world of defining the “grey’ in our universe!

  • Right up front, “I also hate to be so negative or contrarian but, this is a NO spin zone and facts are its product; I can always be WRONG but … I am mostly EARLY!”


Opinions expressed are those of the author and are subject to change, and not intended to be a forecast of future events, a guarantee of future results, nor investment advice. Whether information or intelligence is good, bad, or somewhere in between; I put into context what is relevant and useful for investors.  All investments are subject to risks. Investors should consider investment objectives.

Henry McCusker, the editor, and publisher of RegMed Investors does not hold or have positions in securities referred to in this publication. Regulation Analyst Certification (Reg AC): The research analyst primarily responsible for the content of this report certifies the following under Reg AC: I hereby certify that all views expressed in this report accurately reflect my personal views about the subject company or companies and its or their securities.

I also certify that no part of my compensation was, is or will be, directly or indirectly, related to the specific recommendations or views expressed in this report.