May 17, 2022 7:38am

The cell and gene therapy sector attempts a demonstration of value appreciation

News: Applied Genetic Technologies (AGTC closed +$0.018, pre-open +$0.03) reported positive three (3) month interim data from its ongoing P2 Skyline trial of AGTC-501, a recombinant AAV viral vector-based gene therapy targeting mutations in the RPGR gene in patients with X-linked retinitis

Pre-open indications: 7 Positive Indications and 1 Puff/Pump/Promote

RMi outlines a daily bell ringing of “indications” about what you need to know; it’s what could happen or materialize after the opening and what might happen during the session.

Just the facts … I ask questions and note the indications of what could happen …


Remember that overnight and pre-open actions in futures don't necessarily translate into actual trading in the coming day’s session.


Dow futures are UP +1.27% (+408 points), S&P futures are UP +1.64% (+65 points) and NASDAQ futures are UP +2.05% (+250 points) early in the pre-open – so far,


Futures are looking very green i.e., sharply up anticipating a positive open on Tuesday,

European stocks advanced,

Asia-Pacific stocks were higher



Tuesday’s gains are about to come after a strong overnight session in Asia encouraged by Covid news out of China.

The Nasdaq underperformed finishing Monday -1.2% lower and is now 28% below its intraday all-time high from 11/22/21 as the bloodbath in cell and gene therapy stocks continued.

Economic Data Docket: retail sales numbers hitting at 8:30 a.m. ET followed by industrial production numbers later in the morning.

News (continued): AGTC – there were re robust improvements in visual sensitivity, the trial’s primary efficacy endpoint, in multiple patients three months after dosing, with a 62.5% response rate in dose group B and a 25% response rate in dose group A. This is well above the statistically significant 50% response rate the Vista Phase 2/3 trial for XLRP is powered to detect.

Coming sector attractions for RegMed companies: Agios (AGIO) plans to reduce up to 50 roles focused on exploratory research, while retaining a strong internal research team focused on roles critical to advancing the company’s current and future late-stage research and early clinical programs. The initiative is expected to deliver annual average cost savings of $40-50 million associated with research and related expenses between 2023 and 2026. How many others are due for an “evolution”?


RegMed Investors’ (RMi) closing bell: “up, down, up around and down settling with a negative sector of cell and gene therapeutics. The issue is and will be pricing sustainability … from Friday’s evening post, I STILL believe we are only “renting” the upside? SELL into Strength was my daily mantra – build cash positions as 52-week lows become “relevant” to portfolio “wallets” …

Ebb and flow –

Q2/22 –

May - 5 positive and 5 negative closes

·         April - 1 holiday, 6 positive and 13 negative closes


·         March – 13 negative and 10 positive closes

·         February stats: 11 negative, 8 positive closes and 1 holiday

·         January stats: 2 holidays, 1 neutral, 13 negative and 6 positive closes


RegMed Investors (RMi) - Q1/22 Cell and Gene Therapy Earnings Scorecard Results … -- added Brainstorm Cell Therapeutics (BCLI), Homology Medicine FIXX) and Biostage (BSTG)


Companies in my headlights – It’s your decision; I provide ideas and context: INDICATIONS

Positive Upside Indication:

Alnylam Pharmaceuticals (ALNY) closed down -$1.60 to $124.87 <52-week low of $116.78>after Friday’s +$5.14 after Thursday’s +$0.91, Wednesday’s -$7.76, Tuesday’s +$5.66 and Monday’s -$12.23 with a positive +$0.43 or +0.34% pre-open indication,

Beam Therapeutics (BEAM) closed down -$2.91 to $32.54 <52-week low of $27.77> after Friday’s +$4.01 to $35.45 after Thursday’s +$1.48, Wednesday’s -$5.82, Tuesday’s +$2.49 and last Monday’s -$2.39 with a negative -$0.04 or -0.12% pre-open indication. Won’t be left behind …

CRISPR Therapeutics (CRSP) closed down -$0.69 to $51.54 <52-week low of $> after Friday’s +$5.69, Thursday’s +$2.87, Wednesday’s -$3.64 and last Tuesday’s +$4.08 following the previous Monday’s -$4.78 with a positive +$1.31 or +2.54% pre-open indication,

Editas Medicine (EDIT) closed down -$0.73 to $11.14 <52-weeek low of $9.71 and a high of $73.03> after Friday’s +$1.06 to $11.87, Thursday’s +$0.78 with a positive +$0.12 or +1.08% pre-open indication,

Intellia Therapeutics (NTLA) closed down -$2.64 to $45.96 <52-week low of $38.85 and a high of $202.73> after Friday’s +$6.00, Thursday’s +$2.66, Wednesday’s -$4.46and last Tuesday’s +$2.95 following the previous Monday’s -$6.12 with a positive +$2.77 +$6.03% pre-open indication,

Ionis Pharmaceuticals (IONS) closed up +$0.52 to $36.60 <52-week low of $25.04 and a high of $44.42> with a positive +$0.40 or +1.09% pre-open upside indication,

Sangamo Therapeutics (SGMO) closed down -$0.14 to $3.88<52-week low of $3.48and a high of $12.83> with a positive +$0.22 or +5.67% pre-open upside indication,


Puff/Pump and Promote: Maintaining SELL

Biostage (OTCQB: BSTG) closed flat again after reporting earnings post Friday’s flat (it was down -$0.89) with 1,811 shares traded, Thursday’s and Wednesday’s 0 shares traded and last Tuesday’s flat with 1,845 shares traded.

  • WARNING:  Earnings reported a VERY SAD picture of a company’s status without U.S investors and reliance on non-U.S. Chinese investors. Another PPM -private placement – this one for $5.1 M without a price per share notification. Also, the terminal death lawsuit just got settled (4/28) for $6 M with a PPM – a preferred convertible debt financing.  is it the ONLY avenue to finance utilizing non-U.S. Chinese investors?


The BOTTOM LINE: The sector could be attempting a comeback from intense selling last week as stock futures surged in pre-market trading Tuesday.

Q1/22 earnings season is more than completed (31 of 35 reported) having reported LPS (loss-per-share) results so far MISSING estimates and revenues.

A show of expression – one (1) net income reported – Homology Medicine (FIXX) reported on Monday

I try to keep it simple … and short!

I caution investors that the severe share pricing’s – technical and fundamental damage has been suffered these past several months which will take longer than a few good sessions to repair.


Whether information or intelligence is good, bad or somewhere in between; RMi’s daily report is a primer that may say little or a lot; yet it serves as insurance that all indications are being examined and evaluated.


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.

All investments are subject to risks. Investors should consider investment objectives.

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.

Henry McCusker, the editor and publisher of RegMed Investors does not hold or have positions securities referred to in this publication.