February 7, 2024 5:18pm

Pre-open indication: 5 Hit and 1 Miss

News: REGENXBIO (RGNX +$1.52) announced topline results from the P1/2/3 CAMPSIITE® trial of RGX-121 for the treatment of patients up to 5 years old diagnosed with Mucopolysaccharidosis Type II (MPS II), also known as Hunter syndrome, demonstrating that the pivotal phase of the trial met its primary endpoint with statistical significance. 

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A NO 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!


Wednesday: The Dow closed UP +156.00 points or +0.40%, the S&P closed UP +40.83 points +0.82% while the Nasdaq closed UP +147.65 points or +0.95%



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 Wednesday, stocks rallied as investors weighed a fresh batch of strong corporate profits and major technology giants continued their march higher.

Economic Data Docket: a big day for Fed speakers, virtually all of whom said they aren’t ready to start cutting interest rates yet and will move slowly once they are.


From title: MPS II, or Hunter Syndrome, is a rare, X-linked recessive disease caused by a deficiency in the lysosomal enzyme iduronate-2-sulfatase (I2S) leading to an accumulation of glycosaminoglycans (GAGs), including heparan sulfate (HS) in tissues which ultimately results in cell, tissue, and organ dysfunction, including in the central nervous system (CNS). MPS II is estimated to occur in 1 in 100,000 to 170,000 births. In severe forms of the disease, early developmental milestones may be met, but developmental delay is readily apparent by 18 to 24 months. Specific treatment to address the neurological manifestations of MPS II remains a significant unmet medical need.


Wednesday’s RegMed Investors’ (RMi) pre-open: “do your best with what you got. Have we pumped the pot for the upside or the end of share pricing growth.”https://www.regmedinvestors.com/articles/13318


Pre-open Indications: 5 Hit < Ultragenyx Pharmaceuticals (RARE -$1.18), Beam Therapeutics (BEAM -$0.31), Blueprint Medicine (BPMC -$2.97), Fate Therapeutics (FATE $0.18), Regenxbio (RGNX +$1.52)> 1 Miss < Vericel (VCEL +$0.07)


Advance/Decline (A/D) Line:

  • Wednesday’s advance/decline line ended with a negative close of 8 incliners, 27 decliners and 0 flat


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

Q1/24: February, 3 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:

  • Wednesday, the IBB was down -1.78% and the XBI was down -1.88%

The CBOE Volatility Index (VVIX: INDEX) tracked:

  • Wednesday was down -0.23 point or -1.76% at 12.08


Wednesday’s Closing Down (10 of 27):

  • Alnylam Pharmaceuticals (ALNY -$3.58 after Tuesday’s +$1.48 and Monday’s -$2.36),
  • Blueprint Medicine (BPMC -$2.97),
  • Ultragenyx Pharmaceuticals (RARE -$1.18 after Tuesday’s +$1.13 and Monday’s +$1.74)
  • CRISPR Therapeutics (CRSP -$1.16 after Tuesday’s +$2.37 and Monday’s -$0.24
  • Sage Therapeutics (SAGE -$0.81),
  • Verve Therapeutics (VERV -$0.51 after Tuesday’s +$0.65 and Monday’s -$0.20
  • Intellia Therapeutics (NTLA -$0.50 after Tuesday’s +$1.73 and Monday’s -$0.45
  • Caribou Biosciences (CRBU -$0.36),
  • uniQure NV (QURE -$0.35),
  • Beam Therapeutics (BEAM -$0.31 after Tuesday’s +$1.95 and Monday’s +$0.01

Wednesday’s Closing Up (8 of 8):

  • Regenxbio (RGNX +$1.52 after Tuesday’s +$0.73 and Monday’s +$0.08)
  • Ionis Pharmaceuticals (IONS +$0.42 after Monday’s-$0.32),
  • Adverum Biotechnologies (ADVM +$0.31 after Tuesday’s +$0.59 and Monday’s +$0.49)
  • Graphite BIO (GRPH +$0.16),
  • Vericel (VCEL +$0.07 after Tuesday’s +$0.94 and Monday’s +$0.33)
  • Voyager Therapeutics (VYGR +$0.06),
  • Cellectis SA (CLLS +$0.01),
  • Homology Medicine (FIXX +$0.0031),


Q1/24 – February

  • Wednesday closed negative with 8 incliners, 27 decliners and 0 flat


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

  • Bonds were under modest pressure, as comments from Fed officials reaffirmed expectations that the central bank may not soon cut rates.
  • The yield on benchmark 10-year Treasury notes rose to 4.0999% from 4.092% previously. The two-year yield, which rises with traders' expectations of higher Fed fund rates, touched 4.3932%, versus its Tuesday close of 4.408%.

Volume was lower on the Nasdaq compared with Tuesday as decliners outnumbered advancers by less than 2-to-1.

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.

 WHY do I keep analyzing Harvard Apparatus GN (HRGN) formerly, Biostage (BSTG):

  • I WAS there to experience the lack of corporate and investor prerogatives, saw the decay of transparency and more; it takes courage, resolve and patience to stay the course of asking the questions without response!
  • Have you notice how it barely trades unless volume is “pumped”; is it by DST Capital, run by the president of HRGN, Hong Yu and Mrs. Mrs. bin Zhao of Weston, Mass – funded by Lui Dong Hai, chairman of Dixintong Technology Group (D. Phone, the largest smartphone retailer in China) the husband of Mrs. bin Zhao?
  • Still labeled a … Going Concern… flailing for the lack of U.S. investors and a VERY short “runway” until Q4/2023’s end … and the back story to Netflix: “Bad Surgeon, Love under the Knife”.

The top three (3) performing in the session:

  • Wednesday: Regenxbio (RGNX), Ionis Pharmaceuticals (IONS) and Adverum Biotechnologies (ADVM)
  • 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:  

  • Wednesday: Alnylam Pharmaceuticals (ALNY), Blueprint Medicine (BLMC) and Ultragenyx Pharmaceuticals (RARE)
  • 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.