September 24, 2025 5:09pm

… While a love-up cell and gene therapy sector appreciates with a sudden and exceptional catalyst

Although caution needed ahead of econs before jobless claims data Thursday and PCE inflation data Friday.

News: UniQure NV (QURE +$33.84 or +247.73% to $47.50) stock catapulted today after its experimental gene therapy successfully treated Huntington's disease. <see below>

Never leave an investor uninformed

News (continued): QURE’s Huntington's disease is a neurological condition that slowly destroys brain cells, robbing patients of the ability to move and reason. But a high dose of QURE's AMT-130 slowed disease progression by 75% after 3 years

 

My insights are conveyed with a simplicity of language to highlight what is informative.

Remember, “The sector is what it is, until it isn’t, even if it doesn’t seem to be, it yet will be.” 

I see myself as a better daily fact collector and my responsibility is to lead investors to interpreted data as I bridge the gap to an investment decision.

 

Wednesday’s RMi pre-open: Stalled or mauled … https://www.regmedinvestors.com/articles/14120

Updated … RMi Research Note: Harvard Apparatus GT (OTCQB): Who is wasting lipstick on this pig? … https://www.regmedinvestors.com/articles/13812

 

Wednesday: The Dow closed DOWN -171.50 points or -0.37%, the S&P closed DOWN -18.95 points or -0.28% while the Nasdaq closed DOWN -75.618 points or -0.34%

  • Theme of the session: market slops as pressures i.e., selling arose

Henry’omics: We need to more than consider the economic environment to comprehend the micro re “our” universe of cell and gene therapy

  • Sales of new single-family homes saw gains in August. In August, new home sales rose 20.5% to 800,000, more than the previous period’s revised reading of 664,000; better than the 650,000 that economists were looking for.

Wednesday’s (my) 40-company covered sector’s advance/decline line opened positive with 33 incliners, 6 decliners and 1 flat ending with a positive close of 28 incliners, 11 decliners and 1 flat

Metrics:  Wednesday, the IBB was down -0.85%, the XBI was up +0.67% while the VIX was down -0.43 points or -2.58% at 16.21

 

Q3 – 1 holiday, 8 negative and 9 positive closes

  • August – 12 negative and 9 positive closes
  • July – 1 market holiday, 13 positive and 9 negative closes

 

Wednesday Closing UP (10 of 28)

  • uniQure NV (QURE +$33.84),
  • Alnylam Pharmaceuticals (ALNY) closed +$3.96 after Tuesday’s -$4.61 after Monday’s +$5.36),
  • BioNTech (BNTX +$1.48),
  • Ionis Pharmaceuticals (IONS +$1.37 after Tuesday’s +$1.21),
  • Solid Biosciences (SLDB +$0.91 after Tuesday’s +$0.16),
  • Intellia Therapeutics (NTLA +$0.68),
  • Regenxbio (RGNX +$0.61),
  • Lenz Therapeutics (LENZ closed +$0.51 after Tuesday’s -$0.73 after Monday’s -$1.01),
  • Ultragenyx Pharmaceuticals (RARE +$0.36 after Tuesday’s -$1.07 after Monday’s +$1.34),
  • Editas Medicine (EDIT +$0.36 after Tuesday’s +$0.01),

Flat (1)

  • Harvard Apparatus RT (OTCQB: HRGN)

Wednesday’s Closing DOWN (11 of 11):

  • IQV Holdings (IQV -$5.21 after Tuesday’s -$1.38 after Monday’s +$0.63),
  • Vertex (VRTX -$3.10 after Tuesday’s -$3.41),
  • Vericel (VCEL -$0.96 after Tuesday’s -$0.60),
  • BioLife Solutions (BLFS -$0.64),
  • Agenus (AGEN -$0.17),
  • Beam Therapeutics (BEAM -$0.14 after Tuesday’s -$0.68),
  • WVE Life Sciences (WVE -$0.10),
  • Mesoblast (MESO -$0.05),
  • MiMedx (MDXG -$0.02),
  • Generation Bio (GBIO -$0.02),
  • Cellectis SA (CLLS -$0.02)

 

The BOTTOM LINE:  More of the … WHYs …

Will econs poison the market’s water of appreciation? As investors look, I say scowl at the Fed's primary gauge for inflation later in the week — the personal consumption and expenditure index.

  • On Friday, the Fed's preferred inflation gauge, the personal consumption and expenditure index for August is expected to tick higher to 0.3% from July and rise 2.7% annually. Core inflation is seen rising 0.2% on a monthly basis and 2.9% annually.

All 3 major indexes suffered back-to-back losses in the wake of hitting new highs Monday.

  • Volume was lower on the Nasdaq compared with the same time on Tuesday.

For the week:

  • The S&P 500 is down 26.39 points, or 0.4%.
  • The Dow is down 193.99 points, less than 0.4%.
  • The Nasdaq is down 133.62 points, or 0.6%.
  • The Russell 2000 is down 13.79 points, or 0.6%.

 

Is a market ripple ahead: Republicans moved forward Friday on their plan to avert a partial government shutdown (9/30 deadline) and up the political pressure on Democrats to accept the GOP approach to a funding deadline looming in less than two weeks.

  • Analyst expectations across the board say this latest round of brinkmanship is more likely to end with a shutdown than recent standoffs that began with gridlock but saw last-minute concessions.
  • Markets are paying little attention so far — a pattern that has recent historical precedence.
  • One possible reason is that government shutdowns since 1995 have had little effect on stocks <IBD>

We seem to be always waiting for … something i.ie., anything

  • As I have written, “Econs have been poison to our universe” … onerous as they have been today Wednesday

 

September: understand the “flow” …

  • 9/24 – Wednesday closed positive with xx positive, x negative and x flat
  • 9/23 – Tuesday closed negative with 7 positive, 32 negative and 1 flat
  • 9/22 – Monday closed positive with 31 positive, 9 negative and 0 flat

Last week:

  • 9/19 – Friday closed negative with 7 positive, 33 negative and 0 flat
  • 9/18 – Thursday closed positive with 33 positive, 6 negative and 1 flat
  • 9/17 – Wednesday closed negative with 10 positive, 30 negative and 0 flat
  • 9/16 - Tuesday closed positive with 29 positive, 9 negative and 2 flats
  • 9/15 - Monday closed negative with 17 positive, 22 negative and 1 flat

 

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

  • “I hate to be so negative or contrarian but, this is a NO spin zone and truth is its product; I can always be WRONG but, I am mostly EARLY!”
  • If I have learned one thing as a former research analyst in a venture and public fund, operating executive, x-FBI, x-military officer and an adjunct (MBA) business school professor now a journalist; my life and your portfolio are largely tied to knowledge of price movements and questioning pricing targets and more.
  • I was once told by a “very” prominent figure early in my career, “write it right, keep it tight without hype and never lose the stride to guide the reader” – they were pretty simple instructions to follow!

 

Why do I keep repeating, so investors can make the connection …

The top three (3) performing in the session:   

  • Wednesday: uniQure NV (QURE), Alnylam Pharmaceuticals (ALNY) and BioNTech (BNTX)   
  • Tuesday: Ionis Pharmaceuticals (IONS), Adverum Biotechnologies (ADVM) and Arrowhead Pharmaceuticals (ARWR)
  • Monday: BioNTech (BNTX) and Intellia Therapeutics (NTLA)

The worst three (3) in the session:

  • Wednesday: IQV Holdings (IQV), Vertex (VRTX) and Vericel (VCEL)
  • Tuesday: Vertex (VRTX), Alnylam Pharmaceuticals (ALNY) and
  • Monday: Lenz Therapeutics (LENZ), Capricor Therapeutics (CAPR) and Adverum Biotechnologies (ADVM)

 

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 it’s 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.