January 12, 2026 5:07pm

With news including licensing deals from AbbVie and Novartis, earnings announcements from Vertex Pharmaceuticals and Alnylam Pharmaceuticals, and regulatory updates from Beam Therapeutics and Atara Biotherapeutics. <read more in The Bottom Line>

No pre-open, as I was and am shuffling back and forth from/in bed by the newest flu

News: Beam Therapeutics (BEAM +$6.14 or +22.29% TO $33.69) highlighting recent updates for its liver-targeted genetic disease and hematology franchises and strategic priorities in 2026, supported by an extended anticipated operating runway. Alignment reached with U.S. FDA on Potential Accelerated Approval Pathway for BEAM-302 in Alpha-1 Antitrypsin Deficiency (AATD). Based on Biomarker endpoints, U.S. Biologics Licensing Application (BLA) Submission for risto-cel (Previously Known as BEAM-101) expected as early as Year-End 2026

RMi collects, curates, interprets and disseminates cell and gene therapy sector (C&GT) fact-based news, trustable and verified intel of share pricing data bridging 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 why! 

I chose to speak-up <even when sick> when many analysts, brokers and commentators have shut-up!

Friday’s RMi Closing Bell: Econs affect a winning session with sector weakness … https://www.regmedinvestors.com/articles/14263  

RMi Research Note: Capricor Therapeutics (CAPR) DUMP before the next big SLUMP! … https://www.regmedinvestors.com/articles/14249      

 

Monday: The Dow closed UP +86.13 points or +0.17%, the S&P closed UP +10.99 points or +0.16% while the Nasdaq closed UP +62,558 points or +0.26%

  • Theme of the session: a risk-off Monday as fed Gov Powell uploads “direct video statement Sunday evening that federal prosecutors have opened a criminal investigation related to his Senate Banking Committee testimony on the renovation of Fed office buildings” <CNBC>

Monday’s (my) 40-company covered sector’s advance/decline line opened negative with 17 incliners, 19 decliners and 4 flats ending with a neutral close of 19 incliners, 19 decliners and 2 flats

The CBOE Fear (VIX) index, Monday closed 15.09, after Friday’s 14.48, Thursday’s 15.47, Wednesday’s 15.34, Tuesday’s 14.77 and last Monday’s 14.91,

Metrics: Monday …

  • The RUT was up +11.47 points or +0.44%,
  • The XLV was up +0.07 points or +0.04%,
  • The NBI was down -42.83 points or -0.74%;
  • The XBI was down -0.57 points or -0.46%
  • The IWM was up +1.27 or +0.49%;
  • The IBB was down -0.99 points or -0.57%,
  • The VIX was up +0.60 points or +4.14% at to 15.09

 

Q1/26 – 1 holiday, 1 neutral, 3 negative and 3 positive closes

Q4/25 …

  • December – 1 holiday, 8 positive and 14 negative closes
  • November – 1 holiday, 8 positive and 11 negative closes
  • October -1 neutral, 11 positive and 12 negative closes

 

Monday Closing UP (10 of 19) 

  • Beam Therapeutics (BEAM +$6.14),
  • BioNTech (BNTX +$2.33 after Friday’s +$1.00),
  • Ultragenyx Pharmaceuticals (RARE +$1.86 after Friday’s -$0.70),
  • CRISPR Therapeutics (CRSP +$1.46 after Friday’s -$2.56),
  • Arrowhead Pharmaceuticals (ARWR +$1.37),
  • Ionis Pharmaceuticals (IONS+$1.36 after Friday’s -$1.24),
  • Intellia therapeutics (NTLA +$1.05),
  • Capricor Therapeutics (CAPR +$0.64 after Friday’s -$1.71 after Thursday’s -$0.76, Wednesday’s +$0.18. Tuesday’s +$0.32 and last Monday’s -$3.50)
  • Regenxbio (RGNX +$0.49),
  • Prime Medicine (PRME +$0.353 after Friday’s +$0.02),

Flat (2)

  • Adverum Biotechnologies ADVM) – acquired
  • Harvard Apparatus RT (OTCQB: HRGN +$0.01 after Thursday’s $0.00)

Monday’s Closing DOWN (10 of 19): 

  • Alnylam Pharmaceuticals (ALNY -$27.38 after Friday’s -$1.11),
  • Vertex (VRTX -$3.27 after Friday’s -$5.82),
  • Sarepta Therapeutics (SRPT -$2.69 after Friday’s +$0.68),
  • Mesoblast (MESO -$2.29 after Friday’s +$1.09),
  • uniQure NV (QURE -$2.28 after Friday’s +$2.17),
  • Vericel (VCEL -$1.97 after Friday’s -$2.17),
  • AxoGen (AXGN -$1.96 after Friday’s +$0.14),
  • IQIA Holdings (IQV -$1.55 after Friday’s -$0.75),
  • BioLife Solutions (BLFS -$0.82),
  • Moderna (MRNA -$0.46 after Friday’s +$0.44),

 

The Bottom Line: More of the … WHY

  • This new subclade K … flu S_c_s!  I just got over covid …! And I got ALL the SHOTS

Indexes notched yet another fresh records on Monday, as all reached new highs …

Also, a lot of talk about after US prosecutors opened a criminal investigation into Fed Chair Powell US after US prosecutors opened an “criminal” investigation into Chair Powell. I say a political hack and future criminal.

  • An unsettled mood crawls as investors get ready for this week's updates on inflation in December, with the Consumer Price Index (CPI) reading due tomorrow.

 

JPM news:

AbbVie is paying Yantai, China-based RemeGen $650 million up front, and potentially more than $5 billion overall, for a bispecific antibody drug targeting the proteins PD-1 and VEGF.

  • The deal announced Monday hands AbbVie rights to develop, manufacture and commercialize RemeGen’s RC148 outside of China and nearby territories.
  • The drug being evaluated in a P1 lung cancer study and Phase 2 trials in other solid tumors and breast cancer. Some of those mid-stage studies are evaluating RC148 alongside an antibody-drug conjugate Pfizer gained rights to through a buyout of Seagen.
  • That ADC is approved in China, as is an autoimmune drug RemeGen licensed to Vor Biopharma last year. — Jonathan Gardner

 

Alnylam Pharmaceuticals (ALNY)  on Sunday pre-announced Q4 results and issued revenue guidance for the coming year. Alnylam said its closely watched transthyretin amyloidosis drug Amvuttra generated $827 million between October and December, which missed consensus estimates by more than $20 million.

  • But the $4.4 billion to $4.7 billion in projected 2026 net revenue from its TTR drugs — which include Amvuttra as well as an older medication, Onpattro — came in ahead of analysts’ projections.
  • The numbers were issued alongside a new 5-year strategy that involves hitting at least a 25% average annual growth rate through the end of 2030.
  • Shares ticked down about 8%. — Ben Fidler

 

Vertex Pharmaceuticals (VRTX) also issued a business update that included insight into the recent sales performance for its pain drug Journavx.

  • According to Vertex, more than 500,000 prescriptions have been filled since Journavx’s approval in January, and the company expects that number to more than triple in the coming year.
  • Vertex has also secured coverage in acute pain with all of the large national pharmacy benefit managers. The updates suggest “steady growth” but imply Q4 sales could come in slightly below consensus estimates of $33 million, wrote RBC Capital Markets analyst Brian Abrahams.
  • Vertex also pre-reported $100 million in revenue for its gene editing treatment Casgevy.
  • That number would come in slightly ahead of Wall Street projections, but the program “remains of low investor interest,” Abrahams wrote. — Ben Fidler

 

Beam Therapeutics (BEAM), meanwhile, said that it's aligned with U.S. regulators on a potential accelerated approval submission for one of its base editing therapies.

  • Beam is testing the therapy in people with the rare lung disease alpha-1 antitrypsin deficiency and, last year, delivered early results suggesting the treatment could correct the disease-causing mutation underlying the condition.
  • On Sunday, Beam said that the FDA could clear the drug, BEAM-302, based on positive effects on surrogate markers over the course of a year.
  • Beam expects to enroll about 50 more patients in an ongoing trial and treat them with the "optimal" dose, and will report updated results, as well as next steps for the program, by the end of the first quarter.
  • Beam additionally anticipates seeking approval of a sickle cell therapy, risto-cel, later this year. Company shares climbed more than 25%. — Ben Fidler

 

Novartis )NVS) is paying $165 million in upfront cash to license an experimental Alzheimer’s disease treatment from SciNeuro Pharmaceuticals.

  • The drug is designed to remove toxic amyloid deposits from the brains of people with Alzheimer’s, but with the help of a “shuttle” technology that helps the medicine cross the blood-brain barrier
  • Multiple other companies, including Roche, are evaluating similar approaches.
  • SciNeuro, which recently raised $53 million in venture funding, could receive another $1.5 billion in the deal. — Jonathan Gardner

 

The FDA has again rejected a cell therapy from Atara Biotherapeutics, the company said Monday.  A year ago, the agency cited manufacturing issues in turning back Atara’s request to approve Ebvallo for a type of post-transplant disease associated with Epstein Barr virus infections.

  • Now, however, Atara said that the agency has reversed its previous position and claimed that the single-arm study supporting the company’s application is no longer sufficient to support an approval. Atara had previously aligned with FDA leaders on the study’s design through “multiple, documented meetings” over the last five years.
  • Partner Pierre Fabre Pharmaceuticals, which holds commercial rights to Ebvallo, intends to meet with the FDA to find a path forward. Atara shares fell by more than 50%. — Delilah Alvarado

 

For all my … <some say> … RANTINGS, I say TRUTH … Capricor Therapeutics (CAPR) closed Monday closed up +$0.67 after being DOWN Friday -$1.71 or -6.69% to $23.84 after Thursday’s -$0.76 or -2.89% to $25.55 after Wednesday’s +$0.20 …

  • CAPR’s I-Banks seemed to be executing the “PUMP” to protect the offering share pricing – LOT of daily volume, institution driven … DON’T be distracted – Make a buck, CONTINUE the SELL of the overbought.
  • Right again after again and yet another again … downward!

 

The recent profit-taking could also foreshadow some of the volatility ahead and …

  • RISK is always a factor remaining and … will boil-up under the surface until or as slight shocks rock the C&GT sector and markets!

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!

 

January – 3nd week:

  • 1/12 – Monday closed neutral with 19 incliners, 19 decliners and 2 flats

January 2nd week

  • 1/9 - Friday closed negative with 10 incliners, 28 decliners and 2 flats
  • 1/8 – Thursday closed negative with 7 incliners, 30 decliners and 3 flats
  • 1/7 – Wednesday closed positive with 33 incliners, 4 decliners and 2 flats
  • 1/6 – Tuesday closed positive with 27 incliners, 11 decliners and 2 flats
  • 1 /5 - Monday closed negative with 18 incliners, 21 decliners and 1 flat

January’s 1st week:

  • 1 /2 - Friday closed positive with 18 incliners, 17 decliners and 5 flats
  • 1/1 -Thursday was a holiday, New Years Day

 

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!

 

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

The top three (3) performing in the session:    Winners

  • Monday: Beam Therapeutics (BEAM), BioNTech BNTX) and Ultragenyx Pharmaceuticals (RARE
  • Friday: uniQure NV (QURE), Mesoblast (MESO) and BioNTech (BNTX)
  • Thursday: Mesoblast (MESO), AxoGen (AXGN) and Intellia Therapeutics (NTLA)
  • Wednesday: Alnylam Pharmaceuticals (ALNY), Vertex (VRTX) and IQVIA Holdings (IQV),
  • Tuesday: Vertex (VRTX), Alnylam Pharmaceuticals (ALNY) and Arrowhead Pharmaceuticals (ARWR)
  • Last Monday: IQVIA Holdings (IQV), BioLife Solutions (BLFS), and Vericel (VCEL)

The worst three (3) in the session: Losers

  • Monday: Alnylam Pharmaceuticals (ALNY), Vertex (VRTX) and Sarepta Therapeutics (SRPT)
  • Friday: Vertex (VRTX), CRISPR Therapeutics CRSP) and Vericel (VCEL)
  • Thursday: Alnylam Pharmaceuticals (ALNY), Vertex (VRTX) and Moderna (MRNA)
  • Wednesday: Arrowhead Pharmaceuticals (ARWR), BioLife Solutions (BLFS) and LENZ Therapeutics (LENZ)
  • Tuesday: AxoGen (AXGN), Ultragenyx Pharmaceuticals (RARE) and Entrada Therapeutics (TRDA)
  • Last: Monday: Capricor Therapeutics (CAPR), Vertex (VRTX) and Arrowhead Pharmaceuticals (ARWR)

 

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.