20 BARRON’S September 30, 2019
invest in data extraction, whether through elec-
tronic medical records, or EMRs, or genomic
data from large sequencing programs and clinical
genetic testing. Companies like Verana Health,
which counts GV as an investor, are starting to
license, organize, and analyze patient and disease
data across multiple disease categories. One area
in which Verana is making a big push is neurol-
ogy, where organizing and examining data might
enable us to break dementia into many different
disease categories, as we have done with cancer.
This opens the door to more precision-based
drug-development strategies, which have a
substantially higher likelihood of success.
Amusa: If we can break larger diseases into
smaller ones—for example, by genetically defin-
ing them—we can start to develop precision ther-
apies. A disease like inflammatory bowel disease,
or IBD, is probably going to be considered some-
thing like 20 or 30 different diseases in 10 or 20
years. If you pick the right therapy for each of
them, you will see better outcomes. In Parkin-
son’s disease, Prevail Therapeutics [PRVL] is
testing a gene therapy targeting a particular
genetically defined population. I don’t cover the
company, but to me that’s a good starting point.
Casdin: The promise of gene therapy to cure rare
genetic disease is now a reality, and yet for most
patients it takes a multiyear diagnostic odyssey
that costs tens of thousands of dollars before they
know what’s wrong with them. We’re investors in
Invitae [NVTA], a company that provides clinical
sequencing for inherited genetic diseases. Invitae
solves the diagnostics problem with a DNA
sequencing-based test costing an average of $
that takes less than two weeks to come back with
an answer as to the genetic mutation responsible.
It isn’t hard to imagine that in 10 years, most chil-
dren, particularly those displaying signs of a rare
disorder, will be sequenced at birth. The potential
of this practice to change clinical care is huge.
Schenkein: Take it a step further. Imagine the
day when you visit your primary-care doctor, who
tells you your blood pressure, cholesterol level,
and germline genome. Based on that information,
you can discuss the diseases you’re at risk for
and therapies that can intervene. This isn’t so far
away—maybe 10 years-plus—given how cheap
sequencing has become, and how much better
we’re getting at understanding the data.
Casdin: Although a huge investment is happen-
ing in data management, most medical records
remain isolated within your individual provider.
If you see a primary-care physician and three
specialists in New York, they’re all using differ-
ent record-keeping systems, and the data are
trapped within each. We have to get much bet-
ter at aggregating large data sets and using
machine-learning tools to understand them.
That is coming—and it’s coming fast.
Let’s move on to the companies that look most
promising to all of you. Gena, care to start?
Wang:Adverum Biotechnologies [ADVM] is
one of the stocks that sank this morning, after
the company disclosed disappointing results in a
Phase 1 study of its gene therapy for wet age-
related macular degeneration. Patients didn’t
see an improvement in visual acuity. We don’t
cover the stock, but we follow it closely. Our
pick is Regenxbio [RGNX], which has a com-
peting gene therapy that appears to reduce the
need for intraocular injections of a monoclonal
antibody, the standard treatment for this incur-
able condition. Before this morning, I was a bit
worried about competitors’ data.
Regenxbio holds the intellectual property
rights for many AAV vectors [adeno-associated
viral vectors used to deliver genes and gene-ed-
iting tools to the patient’s body], including the
vector licensed by AveXis to develop Zol-
gensma. The value of thisprovides a floor value
for the stock. Regenxbio hasn’t benefited much
yet from the Zolgensma launch, but we expect
that it will. In the fall, we will have more data
on Regenxbio’s wet AMD gene therapy, which
could provide upside to the stock. Our price
target is $88. Regenxbio is trading for around
$40 a share.
Sarepta Therapeutics [SRPT] is another
gene-therapy company we like. The stock is out
of favor, having fallen on negative news. Sarepta
focuses on treatments for Duchenne muscular
Picks
Vertex ...................................
Pharmaceuticals
VRTX
$169.
Ascendis Pharma.........................
ASND
$95.
Tricida ...................................
TCDA
$30.
Ziad Bakri
Portfolio Manager
T. Rowe Price Health
Sciences Fund
Note:Pricesasof9/26/
Source: Bloomberg
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