August 22, 2022 4:40pm

More frequently right than consequentially wrong

News: Verve Therapeutics (VERV -$0.72) new pre-clinical data of a second product candidate, VERVE-201. VERVE-201 is designed to permanently turn off the ANGPTL3 gene in the liver, a key regulator of cholesterol and triglyceride metabolism, with a precise A-to-G base pair DNA change. Pfizer (PFE) and its German partner BioNTech (BNTX) have asked the FDA to authorize updated Covid booster shots that target the omicron BA.4 and BA.5 omicron subvariants. Precigen (PGEN -$0.085) has completed the sale of its wholly-owned non-healthcare subsidiary, Trans Ova Genetics, L.C., an animal reproductive technologies company, to URUS, for $170 million in upfront cash and up to $10 million earn-out based on the performance of Trans Ova in 2022 and 2023.

Pre-open indications: 8 HIT and 1 MISS

WHY and HOW is this market affecting the cell and gene therapy sector … “The Bottom Line” underlines the framework of share pricing actions.  Who else is tracking a broad grouping of cell and gene therapy equities – can you afford to not read facts supported by real numbers?

No tunnel vision here, this analyst/journalist gets into the weeds of daily share pricing and root causes 

If I have learned one thing as a former research analyst, venture and public fund investor now a journalist; it is that your life and your bank account are largely tied to your knowledge of price movements and questioning pricing targets.


The Dow closed DOWN -643.13 points (-1.91%); the S&P closed DOWN -90.49 points (-2.14%) while the Nasdaq closed DOWN -323.64 points (-2.55%)



Major indexes opened more than 1% lower and are trading sharply lower as investors braced for a more hawkish Federal Reserve ahead of Fed Chairman Jerome Powell's speech on Friday.

Market strategists blamed the weakness in stocks on expectations for more aggressive interest-rate hikes.

Volume on the Nasdaq was down 13% compared with the same time on Friday. NYSE also saw 6% lower volume.

Economic Data Docket: Durable goods and home sales data Wednesday; revised Q2 GDP on Thursday and personal expenditure PCE numbers due Friday at 8:30 a.m. ET,

Stand-out Volume:

  • Verastem (VSTM -18.082 M versus 3-month average of 1.731 M)

News (continued):

  • Verve Therapeutics (VERV -$0.72) VERV is initially developing VERVE-201 for the treatment of homozygous familial hypercholesterolemia (HoFH), a rare genetic subtype of atherosclerotic cardiovascular disease (ASCVD) characterized by extremely high blood low-density lipoprotein cholesterol (LDL-C), as well as for patients with ASCVD who have not achieved goal LDL-C with oral therapy and a PCSK9 inhibitor.
  • Precigen (PGEN) has completed the sale of its wholly-owned non-healthcare subsidiary, Trans Ova Genetics, L.C., an animal reproductive technologies company, to URUS, a holding company with cooperative and private ownership, for $170 million in upfront cash and up to $10 million earn-out based on the performance of Trans Ova in 2022 and 2023. PGEN intends to use the proceeds to pay the senior convertible notes.


RegMed Investor’s (RMi) Pre-Open: “the cell and gene therapy sucked pond water most of last week” …


RegMed/Stem/Cell and Gene Therapy’s 35 covered equities’ Advance/Decline (A/D) lines: what happened …

  • Monday’s advance/decline line opened neutral at 16 up/ 16 down and 3 flats, flipped negative with 14 up/20 and 1 flat at the mid-day, ending with a negative close of 7/27 and 1 flat.


Pre-open Indications: 8 HITs < Biostage (BSTG $0.00 with 350 shares traded), BioLife Solutions (BLFS -$0.69), Caribou Biosciences (CRBU -$0.07), Chinook Therapeutics (KDNY -$0.17), CRISPR Therapeutics (CRSP -$2.24), Intellia Therapeutics (NTLA -$2.03), Verve Therapeutics (VERV -$0.72), Vericel (VCEL -$2.45) 1 Miss < Homology Medicine (FIXX -$0.03)>


Key Metrics:

  • Monday - Sector volume was HIGHER with 1 of the 7-upside having higher than the 3-month average volume with LOW volume of 1 of 27-downside having higher than the 3-month average volume;

The iShares NASDAQ Biotechnology (IBB) and the SPDR S&P ETF (XBI) indicators:

  • Monday, the IBB was down -1.60% and the XBI was down -1.51%

The CBOE Volatility Index (VVIX: INDEX) tracked:

  • Monday was up +3.50 points or +16.99% at 4.10


Jumping with share pricing momentum (7 of 7):

  • Alnylam Pharmaceuticals (ALNY +$1.94 after Friday’s -$7.48),
  • Brainstorm Cell Therapeutics (BCLI +$0.25 after Friday’s +$0.015),
  • Global Blood Therapeutics (GBT +$0.20),
  • Voyager therapeutics (VYGR +$0.08),
  • Editas Medicine (EDIT +$0.04 after Friday’s -$1.22),
  • Ionis Pharmaceuticals (IONS +$0.02),
  • Verastem (VSTM +$0.02),
  • Applied Genetic Technologies (AGTC +$0.0071),

Flat (1):

  • Biostage (BSTG)

Closing down (10 of 27):

  • Vericel (VCEL -$2.56 after Friday’s -$0.57),
  • CRISPR Therapeutics (CRSP-$2.25 after Friday’s -$3.55),
  • Intellia Therapeutics (NTLA -$2.05),
  • Beam Therapeutics (BEAM -$1.88 after Friday’s -$4.29),
  • Sage Therapeutics (SAGE -$0.91),
  • Verve Therapeutics (VERV -$0.75 after Friday’s -$2.07),
  • Ultragenyx (RARE -$0.88 after Friday’s +$0.36),
  • BioLife Solutions (BLFS -$0.61 after Friday’s -$1.01),
  • uniQure NV (QURE -$0.50),
  • AxoGen (AXGN -$0.34 after Friday’s -$0.44),


August, Q3/2022:

  • Monday closed negative with 7 incliner, 27 decliners and 1 flat


The BOTTOM LINE: I try to keep it simple … and short!

August is revealing more about the ill health of the cell and gene therapy sector.

The cell and gene therapy sector is STILL experiencing the “flight of Icarus” …  since “our” universe have oscillated between gains and losses since the first of the year!

There are really three camps, electronic algorithms, traders versus investors who STILL don’t comprehend if the worst is behind us and those “entities” that continue to sell into selloffs.

There are ONLY net and per share losses in Q2. SG&A’s are, I believe “OUT-OF-CONTROL”, CEOs are skimming shareholders with HIGH salaries and perks as share pricing gets hammered on alternate sessions.

The real question is how recently have CEOs bought-into their depreciated share pricing such as two: AVRO and AGTC.

As I have “noticed” yet again from to date reporting, “Most reporting sector companies … to date have reported net losses, lagging revenue estimates … although AXGN, FATE, RGNX, FIXX and NTLA beat on revenues generating forward motion while ATM use is up.”

I find it very interesting to evaluate share price over earnings and a shortage of revenue reporting as a proxy for share pricing performance.

I stick to my view of anticipating the risks – as my careers focus has been “warning analysis”.


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.