Update-1 A bit of a sell-off in gene therapy but MGTX at new highs at $29.44.

  • Profit taking on Sarepta down 3.84% to $150.59.
  • Roche (RHHBY) and Merck (MRK) near new highs.
  • Mid-caps mixed but Novacure (NVCR) rally 3.27% toward new highs.
  • One of our top picks Amarin (AMRN) from the JPMorgan Healthcare Conf soared 16.27% on revenue guidance boost. 
  • XBI stalled at $87.62 down 0.71%.

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Gene Therapy Big Winners 2019 YTD

  • Sarepta (SRPT) takes lead in Duchenne’s Muscular Dystrophy (DMD) and a broad portfolio.
  • M&A has already begun with Roche acquisition of Spark Therapeutics.
  • Large biopharma is under fire on pricing and generics and technologies are old.

We have written about gene therapy stocks several times in the past but on March 1, 2019 Barron’s wrote an article : “Why Big Pharma is Diving into Gene Therapy” and this catapulted the clinical stage companies bringing sharper focus to investors.(see companies below).Most of these companies focus on rare diseases and Hemophilia A is a substantial opportunity for a cure. Duchenne  muscular dystrophy (DMD) is another important target for replacement gene therapy and recently Sarepta (SRPT) had competitive news that drove their stock up 17% last Friday.Apparently recent clinical data from Pfizer (PFE) released suggest an edge for Sarepta’s gene therapy because of safety issues.

  1. Voyager Therapeutics (VYGR) up 189%.
  2. MetraGTx (MGTX) up 173%.
  3. Unique (QURE) up 174%
  4. Audentes (BOLD) up 70.45%
  5. CRISPR Theraputics (CRSP) upo 57.68%

 

Selected Gene Therapy Stocks 2019

We hope to add cover  new “gene therapy” stocks at a later date like the recent hot IPO Adaptive Biotechnologies (ADPT) focused on the immune systems and T-cell receptors. Also note that Spark Therapeutics (ONCE) is in the final stages of a $4.8B buyout of Roche (RHHBY) but delayed amid competitive worries.

From Barron’s
(6 stocks below))
Company Symbol Price Price at Mkt Cap YTD Perf
6/29/19 52 wk hi $B Perf %
Audentes Therap BOLD 37.86 42.7 1.67 70.46
MetraGTx MGTX 26.88 27 0.896 173.45
RegenexBio RGNX 51.37 85.1 1.88 18.56
Solid Biosci SLDB 5.75 54.54 0.37 -80
uniQure QURE 78.15 82.49 2.95 174
Voyager Therap VYGR 27.22 28.79 0.612 189
Diversified Plays
Biomarin BMRN 85.65 106.74 15.337 0.81
Sarepta SRPT 151.95 165.87 11.27 41.35
Other
Amicus FOLD 12.48 16.82 3.174 24.3
bluebird bio BLUE 127.2 183.6 7.012 27.84
Gene Editing
CRSPR Therap CRSP 47.1 66.26 2.58 57.68
Editas Medicine EDIT 24.74 38.77 1.22 6
Intellia Therap NTLA 16.37 35.3 0.748 19.49
Sangamo Ther SGMO 10.77 -3.9 1.24 -8.57
IBB 109.1 122.97 11.74
XBI 87.71 101.17 20.38

It is a little late to initiate new positions in gene therapy but every portfolio needs to have 2-3 of these stocks. All of them are volatile and the technologies are at an early stage of clinical development so opportunities will arise.

Disclosure long CRSP MGTX

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