December 30, 2025 4:50pm

Driven down by a succession of tech and healthcare selling in last sessions of December, Q4 and FY25

Right again, just early:  STICKING to my CONVICTION that Capricor Therapeutics (CAPR) is overbought and whose valuation is disconnected from fundamentals. CAPR closed down -$1.08 to $28.96 after Monday’s +$0.07, Friday’s -$0.27 and Wednesday’s -$0.57) …  Once it has begun; timing will be everything until the FDA “speaks;” the higher CAPR rose from 12/2’s $6.36, the steeper the fall! DUMP before the continuous SLUMP!

News: Ultragenyx Pharmaceutical (RARE) has completed the rolling submission of its Biologics License Application (BLA) to the FDA <read below>

Pre-open Signal Results: 3 Hits and 2 Miss

RMi collects, curates, interprets and disseminates cell and gene therapy sector (C&GT) fact-based news, trusted and verified intel and share pricing data to bridge the gap to an investment decision

Never leave a retail investor uninformed!  It’s not always time to buy or sell; but it is time to KNOW and why! I chose to speak-up when many analysts have shut-up!

 

The peril facing the cell and gene therapy (C&GT) sector’s share pricing isn’t just applications of macro or even micro econs, sentiment or algorithms within electronic trading; it’s losing highly reliable, accurate and transparent data.

 

News: Ultragenyx Pharmaceutical (RARE) has completed the rolling submission of its Biologics License Application (BLA) to the FDA seeking approval for DTX401 AAV gene therapy (pariglasgene brecaparvovec) as a treatment for Glycogen Storage Disease Type Ia (GSDIa).

  • About DTX401 (pariglasgene brecaparvovec) DTX401 is an investigational AAV8 gene therapy designed to deliver stable expression and activity of G6Pase under control of the native promoter to allow the treated liver cells to respond to normal hormonal signals intended to manage glucose, including insulin and cortisol.
  • The BLA for DTX401 is based on data from a rigorous clinical development program that includes 52 treated patients and up to six years of follow-up.
  • Previously announced data from the randomized, double-blind, placebo-controlled P3 GlucoGene study demonstrate that patients treated with DTX401 experienced significant and clinically meaningful reductions in both the quantity and frequency of daily cornstarch intake while maintaining low levels of hypoglycemia, improved levels of euglycemia, and improved fasting tolerance.
  • These clinical benefits translated to meaningful improvements in patient-reported quality of life, as measured by the Patient Global Impression of Change (PGIC) scale. DTX401 was well tolerated, with an acceptable safety profile.

 

Today’s session falls within the Santa Claus Rally, a historically strong 7-day stretch (12/24/25 – 1/5/26) for stocks that spans the final days of December and early January with thin trading volumes

Tuesday: The Dow closed DOWN -94.87 points or -0.20%, the S&P closed DOWN -9.50 points or -0.14% while the Nasdaq closed DOWN -55.27 points or -0.24%

  • Theme of the session: coming off a losing session to another

Henry’omics: We need to more than consider the macro-economic environment to comprehend the micro re “our” universe of cell and gene therapy (C&GT) sector

  • The S&P Cotality Case-Shiller U.S. National Home Price’s 20-city composite rose 1.3% year over year in October. Economists polled by FactSet anticipated a 1% rise on the year.

Tuesday’s (my) 40-company covered sector’s advance/decline line opened negative with 4 incliners, 34 decliners and 2 flats ending with a negative close of 9 incliners, 31 decliners and 2 flats

The CBOE Fear (VIX) index, Tuesday closed at 14.36, after Monday’s 14.15, Friday’s 13.60, Thursday was Christmas, Wednesday closed at 13.80 and the previous Tuesday’s 13.95

Metrics: Tuesday …

  • The RUT was down -19.21points or -0.76%,
  • The XLV was down -0.10 points or -0.06%,
  • The NBI was down -59.17 points or -1.03%;
  • The XBI was down -1.72 points or -1.39%
  • The IWM was down -1.85 or -0.74%;
  • The IBB was down -1.87 points or -1.10%,
  • The VIX was up +0.16 points or +1.13% at to 14.36

 

Q4/25 – December – 1 holiday, 8 positive and 13 negative closes

  • November – 1 holiday, 8 positive and 11 negative closes
  • October -1 neutral, 11 positive and 12 negative closes

 

Tuesday Closing UP (7 of 7) 

  • Ultragenyx Pharmaceuticals (RARE +$3.06 after Monday’s -$14.47 and Friday’s -$0.39),
  • IQVIA Holdings (IQV +$0.53 after Monday’s +$0.22 and Friday’s +$0.80),
  • Compass Therapeutics (CMPX +$0.23),
  • BioNTech (BNTX +$0.08 after Monday’s +$0.68 and Friday’s -$0.51),
  • Prime Medicine (PRME +$0.07),
  • Sangamo Therapeutics (SGMO +$0.0187),
  • Solid Biosciences (SLDB +$0.01),

Flat (2)

  • Adverum Biotechnologies ADVM) – acquired
  • Harvard Apparatus RT (OTCQB: HRGN $0.00 after Monday's $0.00 and Friday’s +$0.04 with 50 shares traded),

Tuesday’s Closing DOWN (10 of 31): 

  • Vertex (VRTX -$6.04 after Monday’s -$3.12),
  • Alnylam Pharmaceuticals (ALNY -$1.57 after Monday’s -$1.77),
  • Supernus Therapeutics (SUPN -$1.36 after Monday’s- $0.32),
  • Capricor Therapeutics (CAPR -$1.08 after Monday’s +$0.35 and Friday’s +$0.01).
  • Ionis Pharmaceuticals (IONS -$0.99 after Monday’s +$0.05 and Friday’s -$0.72),
  • Vericel (VCEL -$0.86),
  • CRISPR Therapeutics (CRSP -$0.78 after Monday’s -$1.11 after Friday’s -$1.63),
  • uniQure NV (QURE -$0.78),
  • Moderna (MRNA -$0.74),
  • AxoGen (AXGN -$0.72 after Monday’s +$0.65 and Friday’ s+$0.16),

 

The Bottom Line: More of the … WHYs

Indexes vacillated Tuesday, as trying to regroup after slides continued into the final stretch of 2025.

  • Stuck in the red zone again while trimming share pricings
  • Short-term momentum has essentially been to the downside

As I had also written, cash is king for investors who want to keep some liquidity and avoid having to sell in a down market,

  • I would be following share pricing’s ascension on current biotech, cell and gene therapy sector upsides; so, some profiteering can be “situated” while maintaining a position, less elevated!

Most C&GT sector equities dramatically dived on Tuesday, again on Monday, after Friday negative close following Christmas on Thursday.

From the Fed:  The Fed's December meeting minutes showed a division among FOMC members over their decision to cut rates earlier this month. The minutes show inflation must come down in order for additional decreases to occur.

  • “Most participants judged that further downward adjustments to the target range for the federal funds rate would likely be appropriate if inflation declined over time as expected," the minutes said. <Yahoo Finance>

 

Back from the abyss:  Ultragenyx Pharmaceuticals (RARE) steadied on Tuesday after the stock cratered by 42% the day before following disappointing P3 results for its bone disease drug. RARE was up +$3.06 or +15.52% to $22.78 after crashing -$14.47 after Friday’s -$0.39.

 

What goes up, comes down:  Capricor Therapeutics (CAPR closed down -$1.08 after Monday’s +$0.07, Friday’s -$0.27 and Wednesday’s -$0.57) …  I am STICKING to my CONVICTION that CAPR is overbought and whose valuation is disconnected from fundamentals. 

  • Doubts still abound re: regulatory fragility and marginal HOPE-3 results and valuation concerns remain a key hang-up!
  • CAPR remains a structurally challenged, chronically unprofitable company whose valuation is disconnected from fundamentals.
  • It has begun; timing will be everything!
  • The higher CAPR rises, the steeper the fall!

 

Low and behold:  Large-cap pharma stocks Eli Lilly (LLY) and Johnson & Johnson (JNJ) have logged gains of roughly 40% in 2025, while Pfizer (PFE) and Merck (MRK) have lagged, with a decline of about 6% and a gain of more than 6%, respectively.

  • LLY’s gains, which catapulted its market cap to more than $1 trillion — a first for a health-care stock — have come on the back of its advancements in treating obesity. While its injectable Zepbound has been a massive success, 2026 is likely to bring FDA approval of orforglipron, its pill for weight loss. If all remains on schedule, the drug could be on the market by the second quarter.
  • PFE has lagged after pipeline disappointments, including in the weight loss space. Ultimately, it battled Novo Nordisk (NVO) in a $10 billion bidding war for obesity drugmaker Metsera.
  • But a weight-loss drug isn’t a guarantee for success as NVO’s 40% drop this year demonstrates. Also, it’s not a requirement for success. JN’s gains have come on the back of the improved performance of its medtech business, which has seen new products come to market, and the planned spinoff of its orthopaedics unit. In early 2026, JNJ hopes to submit its Ottava surgical robot for FDA approval. <CNBC>

 

Take it a session at a time … the REAL question is … how long will this sentiment and conviction “run” last in the “clean-out-the-exposure” December?

 

5th and last week of December, Q4 and FY25

  • Tuesday closed negative with 7 positive, 31 negative and 2 flats
  • Monday closed negative with 12 positive, 24 negative and 4 flats

4th week of December:

  • Friday closed negative with 11 positive, 28 negative and 1 flat
  • Thursday was a holiday
  • Wednesday closed positive with 31 positive, 5 negative and 4 flats - not published
  • Tuesday closed negative with 14 positive, 24 negative and 2 flats
  • Monday closed positive with 30 positive, 8 negative and 2 flats

 

As the leading voice of cell and gene therapy investors; I am NOT always a doom and gloom analyst/journalist but a man who has been “there” (BUY, SELL, VC and multiple operating roles) with the gray hair and a readership following with real numbers to prove it!

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 facts and truth are its products; I can always be WRONG but, I am mostly EARLY!
  • if I wanted to be liked, I wouldn’t have been an analyst/journalist.

 

Why do I keep repeating, framed in a different para, so investors can make the connection

The top three (3) performing in the session:   

  • Tuesday: Ultragenyx Pharmaceuticals (RARE), IQVIA Holdings (IQV) and Compass therapeutics (CMPX)
  • Monday: BioNTech (BNTX), AxoGen (AXGN) and Capricor Therapeutics (CAPR)
  • Friday: IQVIA Holdings (IQV), AxoGen (AXGN) and Mesoblast (MESO),
  • Thursday: was a holiday

The worst three (3) in the session:

  • Tuesday: Vertex (VRTX), Arrowhead Pharmaceuticals (ARWR) and Alnylam Pharmaceuticals (ALNY)
  • Monday: Ultragenyx Pharmaceuticals (RARE), Vertex (VRTX) and Alnylam Pharmaceuticals (ALNY)
  • Friday: CRISPR Therapeutics (CRSP) and Moderna (MRNA) and Ionis Pharmaceuticals (IONS)
  • Thursday: was a holiday

 

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.