May 13, 2025 5:19pm

As I wrote this a.m., “Gravity … Is a force causing any 2 bodies to be attracted toward each other; I.e., tops and bottoms.”

News: REGENXBIO (RGNX +$0.21) FDA accepts with Priority Review of the BLA for RGX-121 i.e., accelerated approval for clemidsogene lanparvovec (RGX-121) for the treatment of Mucopolysaccharidosis II (MPS II), also known as Hunter syndrome.  Partner Nippon Shinyaku to lead commercialization upon potential approval for MPS II.  RGX-121 on track to be the first gene therapy and one-time treatment for MPS II

Earnings:

  • Regenxbio (RGNX) Net income of +$6.1 M or +$0.12, collaboration revenue of $89.01 M, cash of $272 M in cash with a 2H/2026 runway.
  • Sangamo Therapeutics (SGMO) Net loss of -$30.6 M or -$0.14, collaboration revenue of $6.4 M, cash -N/A with new financing.
  • Editas Medicine (EDIT) Net loss of -$76.1 M or -$0.92 <-$0.43 adjusted for restructuring>, collaboration revenue of $4.7 M, cash of $221 M in cash with a 2026 runway.
  • Cellectis SA (CLLS) Net loss of -$3.9 M or -$0.32, collaboration revenue of $12 M, cash of $246 M in cash with a 2H/2027 runway.
  • Fate Therapeutics (FATE) Net loss of -$37.69 M or -$0.15, collaboration revenue of $1.6 M, cash of $273 M in cash with a 1H/2027 runway.

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.

 

Tuesday’s RegMed Investors’ (RMi) pre-open: Gravity … https://www.regmedinvestors.com/articles/13916

Monday night’s RegMed Investors (RMi) Closing Bell: A rising tide lift all boats … https://www.regmedinvestors.com/articles/13915

 

RegMed Investors (RMi) Financing: Sangamo Therapeutics (SGMO) prices 12,235 M share offering at $0.50 to raise $23 M … https://www.regmedinvestors.com/articles/13917

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

 

Tuesday: The Dow closed DOWN -269.67 points or -0.64%, the S&P closed UP +42.36 points or +0.72% while the Nasdaq closed UP +301.74 points or +1.61%

  • Indexes split as the &P and Nasdaq clawed upward

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 consumer price index, a broad measure of goods and services costs across the U.S. economy, increased 2.3% on an annualized basis in April.

Tuesday’s advance/decline line opened with a negative 4 incliner, 29 decline and 2 flats ending with a negative close of 6 incliners, 28 decliners and 1 flat      

Metrics:  Tuesday, the IBB was down -2.52%, the XBI was down -1.90% while the VIX was down -0.17 points or -0.92% at 18.22

  • The VIX had soared as high as 60.13 in early April as investors worried that rising trade tensions would knock the economy into a recession.

 

Q2/25 – May – 5 negative and 4 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

 

Tuesdays Closing UP (6 of 6) 

  • Vericel (VCEL +$0.98 after Monday’s +$0.92),
  • Mesoblast (MESO +$0.56),
  • Regenxbio (RGNX +$0.21)
  • Harvard Apparatus RT (OTCQB: HRGN +$0.04 after Monday’s $0.00
  • bluebird bio (BLUE +$0.02 after Monday’s -$0.21),
  • Generation Bio (GBIO +$0.0023)

Flat (1)

  • Homology Medicine (FIXX)

Tuesday’s Closing DOWN (10 of 28): 

  • Alnylam Pharmaceuticals (ALNY -$1.33 after Monday’s +$19.04),
  • Ionis Pharmaceuticals (IONS -$1.00 after Monday’s +$1.27),
  • Blueprint Medicine (BPMC -$0.90 after Monday’s +$3.14),
  • BioLife Solutions (BLFS -$0.80 after Monday’s +$0.60)
  • Ultragenyx Pharmaceuticals (RARE -$0.69 after Monday’s +$0.98),
  • AxoGen (AXGN -$0.51),
  • Adverum Biotechnology (ADVM -$0.495 after Monday’s -$9.19),
  • Lenz Therapeutics (LENZ -$0.41 after Monday’s +$3.03),
  • CRISPR Therapeutics (CRSP -$0.37 after Monday’s +$1.97),
  • uniQure NV (QURE -$0.34),
  • Sangamo Therapeutics (SGMO -$0.2868)

 

The BOTTOM LINE: Earnings, share price burnings and offering; just another session in a sea of alteration

Cell and gene therapy sector 2nd session closed negative having started the 3rd week …with a positive close followed by a negative drop on Tuesday

  • Ending with a Friday Humpty-Dumpty negative fall post Thursday and Wednesday’s positive closes … followed by 2 negative closes (the previous Tuesday and Monday)

What more can be written, a surprise deal to dramatic call cut tariffs

May ‘25: understand the “flow” …

  • 5/13 – Tuesday closed negative with 6 positive, 28 negative and 1 flat
  • 5/12 - Monday closed positive with 39 positive, 3 negative and 2 flats
  • 5/9 - Friday closed negative with 10 positive, 23 negative and 2 flats
  • 5/8 – Thursday closed positive with 25 positive, 8 negative and 2 flats
  • 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

 

I usually write/say “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>

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!

Market news i.e., tariff drive upside yet estimate and consensus misses with continued earnings LPS (loss-per-share) will shake the sector down – the vig must be paid!

“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.

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.
  • Review the SGMO offering ... UGLY!

 

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

The top three (3) performing in the session:   

  • Tuesday: Vericel (VCEL), Mesoblast (MESO) and Regenxbio (RGNX)
  • Monday: Alnylam Pharmaceuticals (ALNY), Blueprint Medicine (BPMC) and Lenz Therapeutics (LENZ)

The worst three (3) in the session: 

  • Tuesday: Alnylam Pharmaceuticals (ALNY), Ionis Pharmaceuticals (IONS) and Blueprint Medicine (BPMC)
  • Monday: bluebird bio (BLUE), Adverum Biotechnologies (ADVM) and Cellectis SA (CLLS)

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.