August 18, 2025 4:42pm

As I wrote this a.m., “hold the line, that is, the Advance/Decline (A/D) line after 4 successive positive closes; now 5

While kicking the dog down the street, a rate cut at Jackson Hole but, the closer it gets, the less likely it looks?

News: Ultragenyx Pharmaceuticals (RARE +$0.01 or +0.03% to 29.70 with a negative aftermarket -$0.19 or -0.64% to $29.51) initiated a rolling submission of a BLA to the FDA seeking approval for DTX401 AAV gene therapy as a treatment for Glycogen Storage Disease Type Ia (GSDIa). RARE has submitted the non-clinical and clinical modules to the FDA and plans to complete the full BLA including submission of the chemistry, manufacturing and controls (CMC) module in Q4/25.

Never leave an investor uninformed!  

“When you’re riding’ ahead of the sector herd, take a look back every now and then to make sure your share pricing “cows” are moving

I think it's important to now take a step back, focus on fundamentals, advancing clinical catalysts, “runways” and recent financings strike

I answer or qualify the sector’s equity’s movement; with an emphasis on which company is gaining or losing stride in their portfolio for the short-term

 

Monday’s RegMed Investors’ (RMi) pre-open: hold the line … https://www.regmedinvestors.com/articles/14067

RegMed Investors (RMi) Research Note: Harvard Apparatus GT (OTCQB): Just another wrinkle – Q2/25’s 10-Q – liabilities exceed cash position … https://www.regmedinvestors.com/articles/13812

 

Monday: The Dow closed DOWN -34.30 points or -0.08%, the S&P closed DOWN -0.65 points or -0.01% while the Nasdaq closed UP +6.797 points or +0.03%

  • Theme of the session, awaiting retailers’ earnings and a later week Fed Chair Powell speech and its meetings’ minutes

Henry’omics: 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

  • The NAHB/Wells Fargo Housing Market Index <a gauge of builder confidence in the market for newly built single-family housing> crept down to 32 this month from 33 in July. That contrasts with expectations the index would rise a little to 34. The reading below 50, which equaled the lowest since December 2022, means builder sentiment had been negative for 16 consecutive months.

Monday’s advance/decline line opened positive with 22 incliners, 10 decliners and 2 flats ending with a neutral close of 18 incliners, 17 decliners and 0 flat

Metrics:  Monday, the IBB was down -0.38%, the XBI was down -0.48% while the VIX was down -0.120. points or -0.80% at 14.97

 

Ultragenyx Pharmaceuticals (RARE) news continued: About DTX401 …

  • DTX401 is an investigational AAV8 gene therapy designed to deliver stable expression and activity of G6Pase-α under control of the native promoter to allow the treated liver cells to respond to normal hormonal signals intended to manage glucose, including insulin, glucagon and cortisol. DTX401 is administered as a single intravenous infusion and has been shown in preclinical studies to improve G6Pase-α activity and reduce hepatic glycogen levels, a well-described biomarker of disease progression.

 

Q3 – August – 6 negative and 6 positive closes

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

Q2/25: June – 1 market holiday, 1 neutral, 9 negative and 11 positive closes, May – 1 market holiday, 10 negative and 11 positive closes and April – 11 positive and 10 negative closes

 

Monday Closing UP (10 of 18) 

  • Alnylam Pharmaceuticals (ALNY +$7.96 after Friday’s +$6.18),
  • IQVIA Holdings (IQV +$1.45 after Friday’s +$1.69),
  • Lenz Therapeutics (LENZ +$0.92 after Friday’s -$3.06),
  • AxoGen (AXGN +$0.56)
  • Generation Bio (GBIO +$0.34 after Friday’s -$0.37),
  • Supernus Therapeutics (SUPN +$0.30),
  • BioLife Solutions (BLFS +$0.16 after Friday’s -$0.98),
  • Fate Therapeutics (FATE +$0.11),
  • Voyager Therapeutics (VYGR +$0.11),
  • MiMedx (MDXG +$0.08 after Friday’s -$0.04)

Flat (0)

Monday’s Closing DOWN (10 of 17): 

  • CRISPR Therapeutics (CRSP -$2.14 after Friday’s +$0.91),
  • BioNTech SE (BNTX -$0.97 after Friday’s +$0.82),
  • Sarepta Therapeutics (SRPT -$0.82 after Friday’s +$1.51),
  • Beam Therapeutics (BEAM -$0.72),
  • uniQure NV (QURE -$0.52),
  • Ionis Pharmaceuticals (IONS -$0.35 after Friday’s +$0.93),
  • Mesoblast (MESO -$0.32),
  • Editas Medicine (EDIT -$0.30),
  • Intellia Therapeutics (NTLA -$0.26),
  • Caribou Biosciences (CRBU -$0.11 after Friday’s -$0.02),

 

The BOTTOM LINE:  The cell and gene therapy sector dived neutral  on Monday as investors having turned their focus to a US-Ukraine meeting, kicking off a week ... soon to be “detonated” by a post Jackson Hole Symposium Fed speech that could define the outlook for interest rates.

  • The release of Fed minutes on Wednesday will set the stage for economic data.
  • Meanwhile, Q2 earnings season is winding down …

Today’s bad: Job cuts continue to climb and pervade the biopharma sector this year, defying hopes that 2024 marked a peak.

  • After shedding more than 14,000 jobs in all of 2024, the industry surpassed 13,000 layoffs by July 2025, a 31% year-over-year increase at the halfway mark. And the tide of pink slips has continued to rise this summer.
  • In August, Merck & Co. laid off 58 employees at its New Jersey site as part of a plan to reduce 8% of its global workforce, which could ultimately put 6,000 people out of a job in the next few years. Bayer, Bristol Myers Squibb, Moderna, Tune Therapeutics and Genentech also announced workforce reductions.
  • As the industry grapples with patent cliffs, stubborn investment and pressure to streamline operations, the upward trend may persist.
  • The driving factors: Layoffs in 2025 are the result of both traditional factors and brand-new ones. “Pipeline rationalization, late-stage trial failures and the shift to bring more manufacturing back to the U.S. are shaping this year’s layoff patterns,” Ashley Finney, senior vice president of business development and recruiting at Bench on Demand, said in an email. Companies face added pressure as drug safety concerns and reduced federal support threaten some programs. <PharmVoice>

My perspective is retail investors should roll-the-dice with econ readings as earnings season kicks-in and momentum wains in the cell and gene therapy sector.

  • NO all ins as historically “our” universe slips and slides as the “season’ resumes; so, hang-in with those with solid partners, lengthened runways and solid cash positions leveraged by solid/stable management teams!

 

Reiterating, the market is … has braced for a historically weak month … August is the worst month for the Dow in data going back to 1988, and the 2nd worst for the S&P 500 and Nasdaq according to the Stock Trader’s Almanac.

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!”

Earnings today: None

  • As earnings slowly come to the last few releases, collaborator revenues and runways has instilled share pricing with forward stability
  • Q2 results so far:  4 net incomes and 25 net losses of 29 releases to date

 

August begins as July ends: understand the “flow” …

  • 8/18 -Monday closed neutral with 18 positive, 17 negative and 0 flat
  • 8/15 – Friday - closed positive with 25 positive, 9 negative and 1 flat
  • 8/14 – Thursday closed barely positive with 18 positive, 17 negative and 0 flat
  • 8/13 – Wednesday closed positive with 32 positive, 2 negative and 1 flat
  • 8/12 - Tuesday closed positive with 28 positive, 6 negative and 1 flat
  • 8/11 – Monday closed negative with 10 positive, 25 negative and 0 flat
  • 8/8 – Friday closed negative with 11 positive, 23 negative and 1 flat
  • 8/7 – Thursday closed negative with 9 positive, 24 negative and 2 flats
  • 8/6 - Wednesday closed negative with 11 positive, 22 negative and 2 flats
  • 8/5 – Tuesday closed negative with 15 positive, 19 negative and 1 flat
  • 8/4 – Monday closed positive with 27 positive, 8 negative and 0 flat
  • 8/1 – Friday closed negative with 9 positive, 20 negative and 6 flats

 

“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:   

  • Monday: Alnylam Pharmaceuticals (ALNY), IQVIA Holdings (IQV) and Lenz Pharmaceuticals (LENZ)
  • Friday: Alnylam Pharmaceuticals (ALNY), IQVIA Holdings (IQV) and Sarepta Therapeutics (SRPT)

The worst three (3) in the session: 

  • Monday: CRISPR Therapeutics (CRSP), BioNTech (BNTX) and Sarepta Therapeutics (SRPT)
  • Friday: Lenz Pharmaceuticals (LENZ), BioLife Solutions (BLFS) and Generation Bio (GBIO)

 

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.