May 7, 2025 4:50pm

As sector share pricing bends, blends and ultimately extends to a barely positive close

With volatility heightening as earnings are released with continued missing of estimates and consensus

Earnings (catching up):

  • Ultragenyx Pharmaceuticals (RARE) Net loss of -$151.08 M or -$1.57, collaboration revenue or $139.3 M, $563 M in cash with a 2027 runway.
  • CRISPR Therapeutics (CRSP) Net loss of -$136M or -$1.58, collaboration revenue or $57.5 M, $1.86 B in cash with a 2027 runway.
  • Voyager Therapeutics (VYGR) Net loss of -$31 M or -$0.53, collaboration revenue or $6.47 K, $295 M in cash with a 2027 runway.

Coming Earnings: BLFS and NTLA  5/8, RGNX on 5/9 and EDIT, CLLS and SGMO 5/12, BCLI 5/13

Never leave an investor uninformed! 

 

On point, short on words, long on facts and being judicious!

My mission is to provide clear, actionable guidance on a regular basis to help investors and traders navigate the uncertain and often irrational financial markets. I put hours of effort to create reporting, while leveraging my 40 years of operating and research experiences to analyze the key market-moving events and distill that into a cogent outlook.

 

Wednesday’s RegMed Investors’ (RMi) pre-open: what went down, comes up… https://www.regmedinvestors.com/articles/13908

Tuesday night’s RegMed Investors (RMi) Closing Bell: Humpty Dumpty … https://www.regmedinvestors.com/articles/13907

 RegMed Investors (RMi) Research Note: Harvard Apparatus GT (OTCQB: HRGN) Chronicles: Yet another "screw job" for/on investors ... "wanting to effect a reverse split of its outstanding common stock" in its recent proxy statement filing!! … https://www.regmedinvestors.com/articles/13812

 

Wednesday: The Dow closed UP +284.97 points or +0.70%, the S&P closed UP +24.37 points or +0.43% while the Nasdaq closed UP +48.50 points or +0.27%

  • Indexes were mixed as Fed signals data is soft and confusing        

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 companies

  • The Fed kept interest rates at the target range of 4.25% to 4.5%; noting “the risks of higher unemployment and higher inflation have risen.”        

Wednesday’s advance/decline line opened with a negative 14 incliner, 17 decline and 4 flats ending with a barely positive close of 16 incliners, 15 decliners and 4 flats     

Metrics:  Wednesday, the IBB was up +0.80%, the XBI was up +0.18% while the VIX was down -1.15 points or -4.64% at 23.60

 

Q2/25 – May – 3 negative and 2 positive closes

  • April – 11 positive and 10 negative closes

Q1/25

  • March, 10 positive and 12 negative closes
  • February – 12 holiday, 11 negative and 7 positive closes
  • January - 2 holidays, 1 market close, 10 negative and 10 positive closes

 

Wednesday’s Closing UP (10 of 16)

  • Alnylam Pharmaceuticals (ALNY +$6.74 after Tuesday’s +$3.74 after Monday’s +$14.16),
  • Vericel (VCEL +$2.02),
  • Ionis Pharmaceuticals (IONS +$1.21),
  • CRISPR Therapeutics (CRSP +$1.09 after Tuesday’s -$4.57 after Monday’s -$0.63),
  • Blueprint Medicine (BPMC +$0.64 after Tuesday’s -$2.67 after Monday’s -$2.81),
  • MiMedx (MDXG +$0.37 after Tuesday’s +$0.03 after Monday’s +$0.30)
  • Beam Therapeutics (BEAM +$0.24 after Tuesday’s -$3.83 after Monday’s -$0.42),
  • Mesoblast (MESO +$0.17),
  • Prime Medicine (PRME +$0.09),
  • Compass Therapeutics (CMPX +$0.07),

Flat (4)

  • Brainstorm Cell Therapeutics (BCLI)
  • Editas Medicine (EDIT)
  • Harvard Apparatus RT (OTCQB: HRGN $0.00 after Friday’s $0.00)
  • Homology Medicine (FIXX)

Wednesday’s Closing DOWN (10 of 15):

  • Ultragenyx Pharmaceuticals (RARE -$1.64 after Tuesday’s -$3.81 after Monday’s -$0.43),
  • uniQure NV (QURE -$1.42 after Tuesday’s -$4.41 after Monday’s +$1.12),
  • Solid Biosciences (BLFS -$0.78),
  • Regenxbio (RGNX -$0.68 after Tuesday’s -$1.84 after Monday’s -$0.64),
  • BioLife Solutions (BLFS -$0.61 after Tuesday’s -$2.25 after Monday’s -$1.07),
  • Lenz Therapeutics (LENZ -$0.55 after Tuesday’s -$3.21 after Monday’s -$0.42),
  • Moderna (MRNA -$0.37 after Tuesday’s -$3.41 after Monday’s +$0.23),
  • Adverum Biotechnologies (ADVM -$0.23),
  • bluebird bio (BLUE -$0.15),
  • Verve Therapeutics (VERV -$0.14),

 

The BOTTOM LINE: Weak, weaker and a … squeak, the cell and gene therapy sector closed positive on reversing from Wednesday, Tuesday and Monday negative after Friday closed positive following Thursday negative close.

My perspective is retail investors should NOT be dipping their toes back into the cell and gene therapy sector until after LPS (loss-per-share) earnings report.

  • NO all ins – just add those with solid partners, lengthened runways and solid cash positions leveraged by solid management teams – there I see losses going forward!

“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 don’t have much to write/say other than “electronic trading and uncle algo” slips in and out, back doors reaping sector disharmony and then pops right back for some profiteering.

  • What can I say, love ‘em, leave ‘em and welcome back to save the sector’s honor – “uncle algo and his electronic dwarfs.” <me>

 

May ‘25: understand the “flow” …

  • 5/7 - Wednesday closed positive with 16 positive, 15 negative and 4 flats
  • 5/6 – Tuesday closed negative with 2 positive, 31 negative and 2 flats
  • 5/5 - Monday closed negative with 9 positive, 23 negative and 3 flats
  • 5/2 – Friday closed positive with 24 positive, 8 negative and 3 flats
  • 5/1 - Thursday closed negative with 14 positive, 19 negative and 2 flats

 

What could 2025 have in store for the capital access space – DESPERATE  

  • "You'll see a number of cell and gene therapy companies DESPARITE drive for financings – they’re desperate for cash, but I do think a lot of cell and gene therapy sector companies NEED to be focused on what is driving their own growth —they're focused on a lot of change BUT … that's happening in pipelines … capital expansion is second.

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

The top three (3) performing in the session:   

  • Wednesday: Alnylam Pharmaceuticals (ALNY), Vericel (VCEL) and Ionis Pharmaceuticals (IONS)
  • Tuesday: MiMedx (MDXG) and Cellectis SA (CLLS)
  • Monday: Alnylam Pharmaceuticals (ALNY), Ionis Pharmaceuticals (IONS) and uniQure NV (QURE)

The worst three (3) in the session: 

  • Wednesday: Ultragenyx Pharmaceuticals (RARE), uniQure NV (QURE) and Solid Biosciences SLDB)
  • Tuesday: uniQure NV (QURE), CRISPR Therapeutics (CRSP) and Beam Therapeutics (BEAM)
  • Monday: Blueprint Medicine (BPMC), BioLife Solutions (BLFS) and Mesoblast (MESO)

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 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.