June 7, 2022 4:41pm

As the cell and gene therapy sector jumps back to its feet

News: bluebird bio (BLUE +$0.63) <below>

Pre-open indications:  5 HITs and 3 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?

It’s a quick read to understand the ebb and flow of the session in one site

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 UP +264.03 points (+0.80%); the S&P closed UP +39.11 points (+0.95%) while the Nasdaq closed UP +113.86 points (+0.94%)



Indexes opened lower but losses evaporated about an hour into the session to drop again around 12:30 p.m. to elevate less than an hour later. This morning's reversal comes after the stock market faded Monday, although it closed higher. The week's action so far reflects the market's volatility.

One retail company, target cutting its quarterly profit margin does NOT forecast a dive to “our’ universe.


Highlights and News today:

bluebird bio (BLUE) announced that the FDA posted briefing documents for the FDA Cellular, Tissue and Gene Therapies Advisory Committee Meeting to review elivaldogene autotemcel (eli-cel) for early active cerebral adrenoleukodystrophy (CALD) in patients without a matched sibling donor and betibeglogene autotemcel (beti-cel) for the treatment of people with β-thalassemia who require regular red blood cell transfusions.

·         The advisory committee meeting will take place June 9-10, 2022. Briefing materials, agendas and webcast information for the meeting can be accessed here. The Company is not responsible for the content of, nor the statements made in, the briefing materials that were prepared by the FDA.

·         The Prescription Drug User Fee Act (PDUFA) goal dates for a decision on approval of beti-cel for people with β-thalassemia who require regular red blood cell transfusions and eli-cel for patients with early active CALD without a matched sibling donor are August 19, 2022, and September 16, 2022.


RegMed Investors’ (RMi) pre-open: “diminished expectation? After a negative sector close and a more than weak aftermarket” …  https://www.regmedinvestors.com/articles/12462


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

  • Tuesday opened positive at 26 up/8 downs and 1 flat, stayed positive at the mid-day to 29/4 and 2 flats, ending with a positive close of 30/3 and 2 flat.


Pre-open Indications: 5 HITs < Biostage (BSTG $0.00 with 65 shares traded), Verve Therapeutics (VERV +$1.18), Chinook Therapeutics (KDNY +$0.87), Mesoblast (MESO $0.00), Editas Medicine (EDIT +$0.90)> 3 MISS < Ultragenyx (RARE +$3.97), Beam Therapeutics (BEAM +$2.63), CRISPR Therapeutics (CRSP +$4.82)>   


Key Metrics: review the disparities between daily sessions …

  • Tuesday - Sector volume was MODERATE with 11 of the 30-upside having higher than the 3-month average volume with HIGHER volume of 2 of 3-downside having higher than the 3-month average volume;

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

  • Tuesday, the IBB was up +2.25% and the XBI was up +5.04%

The CBOE Volatility Index (VVIX: INDEX) tracked:

  • Tuesday was down -1.08 points or -4.31% at 23.99


Jumping with share pricing momentum (10 of 30):

  • CRISPR Therapeutics (CRSP +$4.82 after Monday’s +$0.01),
  • Ultragenyx (RARE +$3.97 after Monday’s -$2.15),
  • Intellia therapeutics (NTLA +$2.91),
  • Beam Therapeutics (BEAM +$2.63 after Monday’s -$1.39),
  • Alnylam Pharmaceuticals (ALNY +$2.38 after Monday’s +$0.11),
  • Fate Therapeutics (FATE +$2.29 after Monday’s -$1.65),
  • uniQure NV (QURE +$1.53),
  • Regenxbio (RGNX +$1.20 after Monday’s -$0.81),
  • Verve Therapeutics (VERV +$1.18),
  • Ionis Pharmaceuticals (IONS +$1.01 after Monday’s -$2.13),

Closing down (3 of 3):

  • Bellicum Pharmaceuticals (BLCM -$0.18 after Monday’s +$0.01),
  • Brainstorm cell Therapeutics (BCLI -$0.05 after Monday’s $0.00),
  • Sage Therapeutics (SAGE -$0.01),


  • Brainstorm Cell Therapeutics (BCLI) and Biostage (BSTG)


June, Q2/2022:

  • Tuesday closed positive with 30 incliner, 3 decliners and 2 flats


The BOTTOM LINE: An altitude reaching session; it seems hard in this continuum to have consecutive days of strength!

I try to keep it simple and short … I don't think we've seen the bottom yet. We haven't had that big sell off yet where we have huge volumes.

… Because there’s so much worry that people use any piece of good news as a chance to sell,”

Reiterating, I believe there will be opportunity in cell and gene therapy equities but, the summer doldrums are still a roadblock. This period of volatility will likely last into the second half of the year.

At the time in June, as consecutive upside sessions are ominous as dramatic upsides after downslides open the door to electronic trading appetites!

What changes … the tug-of-war as electronic trading and algorithms take their piece of the upside and then their toll!

Again, as have I written (repeatedly), “There's a battle in the cell and gene therapy sector between which or what breaks first: share pricing resilience or tumbling into yet another downturn as fear remains beneath the strength of any positive close” …

I am not always right but, more than frequently not wrong … I just DO NOT trust this market … too much fear and panic. This continuing state of volatility confirms fears that I have been expressing in these daily and weekly posts.

Reiterating, “I continue to expose the feeling … “like Sisyphus … eternally to repeatedly roll a heavy rock up a hill … only to have it roll down again as it nears the top … as I watch the roller-coaster go round.

·         Reiterating, “Sisyphus teaches us to never give in to circumstantial disappointments or try to escape from the failures, rather accept failures the same way we accept our achievements. And most importantly, no matter how much we lose in our quest, we must never back down till we fulfill our potential.”

However, the continuing state of volatility confirms fears that I have been expressing these daily and weekly posts.

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.