February 12, 2024 4:50pm

Sentiment control, the cell and gene therapy sector was cleared to a higher altitude to climb; what about tomorrow?

News: Sangamo Therapeutics (SGMO +$0.255) The FDA has agreed in a Type D meeting that data from a single, adequate, and well-controlled study may form the primary basis of approval of a BLA for isaralgagene civaparvovec (or ST-920, its wholly owned gene therapy product candidate for the treatment of Fabry disease). The proposed study would enroll up to 25 patients, both male and female, without the need for a control arm. A head-to-head comparison with Enzyme Replacement Therapy (ERT) is not part of the proposed study design deemed acceptable by the FDA. This approach enables a potentially more rapid, efficient and cost-effective pathway to BLA submission than originally anticipated.

Pre-open Indications: 6 Hits and 1 Miss

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A 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!


Monday: The Dow closed UP +125.60 points or +0.33%, the S&P closed DOWN -4.77 points -0.09% while the Nasdaq closed DOWN -48.12 points or -0.30%



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 closed mixed on Monday awaiting new inflation numbers this week.

Economic Data Docket: CPI, a key inflationary gauge — set to be released on Tuesday morning


Monday’s RegMed Investors’ (RMi) pre-open: Earnings’ season kicks-off. No super bowl flu here, but a week including inflation and consumer spending highlighting the economic condition.”  … https://www.regmedinvestors.com/articles/13324    


Pre-open Indications: 6 Hits: < Sell into Strength: Alnylam Pharmaceuticals (ALNY -$0.69), Blueprint Medicine (BPMC +$1.00), Vericel (VCEL +$0.64), Ultragenyx Pharmaceuticals (RARE +$0.01); Positive Indications: Adverum Biotechnologies (ADVM +$0.26), Regenxbio (RGNX +$1.15), Negative Indication:  1 Miss < Sage Therapeutics (SAGE +$0.14)>


Advance/Decline (A/D) Line:

  • Monday’s advance/decline line at the open was positive with 25 incliners, 9 decliners and 1 flat; ending with a positive close of 28 incliners, 7 decliners and 0 flat


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

Q1/24: February, 3 negative and 5 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:

  • Monday, the IBB was up +0.87% and the XBI was up +2.34%

The CBOE Volatility Index (VVIX: INDEX) tracked:

  • Monday was up +1.00 point or +7.73% at 13.93


Monday’s Closing Down (7 of 7):

  • Ionis Pharmaceuticals (IONS -$1.02 after Friday’s +$1.42),
  • Alnylam Pharmaceuticals (ALNY -$0.69 after Friday’s +$0.53),
  • Graphite Bio (BRPH -$0.05),
  • Cellectis SA (CLLS -$0.03 after Friday’s -$0.03),
  • Brainstorm Cell Therapeutics (BCLI -$0.02 after Friday’s -$0.0169),
  • Harvard Apparatus GN (HRGN -$0.02 with 5,850 shares traded <3-month average = 1,390 shares),
  • Homology Medicine (FIXX -$0.0017 after Friday’s $0.0093

Monday’s Closing Up (10 of 28):

  • CRISPR Therapeutics (CRSP +$6.43after Monday’s +$1.18
  • Beam Therapeutics (BEAM +$4.35 after Monday’s +$0.94
  • Intellia Therapeutics (NTLA +$1.38 after Monday’s +$0.76
  • Regenxbio (RGNX +$1.15 after Monday’s -$0.08
  • Verve Therapeutics (VERV +$1.03 after Monday’s +$0.59
  • Blueprint Medicine (BPMC +$1.00 after Monday’s +$2.38
  • Editas Medicine (EDIT +$0.68),
  • Vericel (VCEL +$0.64 after Friday’s +$0.93
  • Fate Therapeutics (FATE +$0.57),
  • Prime Medicine (PRME +$0.55),


Q1/24 – February

  • Monday closed positive with 28 incliners, 7 decliners and 0 flats


The BOTTOM LINE: Earnings’ week – 4 of them

This week will bring a new challenge to the rally, with the January reading of the Consumer Price Index due Tuesday.

  • The CPI report will give investors their first insight into how cool inflation is running in 2024 and, alongside an update on consumer spending, will set expectations for the timing and pace of Federal Reserve interest rate cuts this year.

Earnings’ LPS (loss-per-share) coming for Blueprint Medicine (BPMC) … the maybes …

  • Blueprint Medicines (BPMC) will report quarterly loss of $2.04 per share in its upcoming release, pointing to a year-over-year increase of 23%. It is anticipated that revenues will amount to $67.28 million, exhibiting an increase of 73.5% compared to the year-ago quarter.
  • Analysts predict that the 'Revenues- Product revenue, net' will reach $64.96 million. The estimate indicates a change of +116.1% from the prior-year quarter.
  • The average prediction of analyst’s places 'Revenues- Collaboration and License Revenue' at $3.00 million. The estimate suggests a change of -65.6% year over year.
  • Analysts expect 'Revenues- Product revenue, net- AYVAKIT/AYVAKYT' to come in at $64.58 million. The estimate indicates a year-over-year change of +114.8%.
  • Shares of BPMC have experienced a change of -7.7% in the past month <from ZACKS>

Coming for Alnylam Pharmaceuticals (ALNY) … maybes …

  • The upcoming report from Alnylam Pharmaceuticals (ALNY) is expected to reveal quarterly loss of $1.20 per share, indicating an increase of 28.6% compared to the year-ago period. Analysts forecast revenues of $443.97 million, representing an increase of 32.5% year over year.
  • The average prediction of analyst’s places 'Product revenues, net' at $339.00 million. The estimate indicates a year-over-year change of +29.6%.
  • The collective assessment of analysts points to an estimated 'Net revenues from research collaborators' of $98.87 million. The estimate suggests a change of +40%
  • Over the past month, ALNY shares have recorded returns of -12.1% <from ZACKS>

Monday:The cell and gene therapy sector had its third positive session in a row.

  • The Nasdaq slipped on Monday afternoon after briefly surpassing its record closing high from November 2021, but the Dow and the S&P 500 rose modestly ahead of two U.S. inflation reports this week that could influence Federal Reserve policy. <Reuters>
  • The small caps Russell 2000 index also surged 1.8%

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 as Sage Therapeutics (SAGE) with Blueprint Medicine (BPMC) on 2/14; Alnylam Pharmaceuticals (ALNY) and Ultragenyx Pharmaceuticals (RARE) on 2/15 announcing Q4 and FY23 which WILL be crucial to continue downside force in the sector as history has taught us i.e., investors.

The top three (3) performing in the session:

  • Monday: CRISPR Therapeutics (CRSP), Beam Therapeutics (BEAM) and Intellia Therapeutics (NTLA)

The worst three (3) in the session:  

  • Monday: Alnylam Pharmaceuticals (ALNY), Ionis Pharmaceuticals (IONS) and Graphite Bio (GRPH)


Harvard Apparatus RN (HRGN): is the pimp, pump and promote of share pricing back? Easy answer ...

  • Question #1, did you notice how it barely trades unless volume is “pump” – 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??


  • Notice how ...HRGN traded -$0.02 with 5,850 shares traded <3-month average = 1,390 shares),


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.