Page 22 - HFA Dateline 2022 Special Edition

“It’s not going anywhere,” agreed However, the therapy does not alter need to prophylactically infuse—or at
Lori Long, institute director for HFA. the genetic code, so people will still least know how to infuse and have easy
be genetic carriers of hemophilia, access to factor, according to Long.
Gene Therapy Promise and able to pass it on to their children. These groups of people could
Limitations Also, the therapy may degrade over include the following:
Here’s why factor is here to stay: time, and there are many questions
about who will be eligible for gene  People with mild and moderate
The idea of gene therapy is that therapy. (See gene therapy article hemophilia A or B. Many
a functional gene is inserted that on page 12.) hemophilia gene therapy clinical
establishes the production of factor trials involve patients with severe
VIII or IX, with the goal of reducing or Numerous gene therapy clinical trials or moderate-to-severe disease.
eliminating the need to get repeated have been going on for decades,  People with hemophilia A or
infusions of factor. but nothing has been commercially B with inhibitors. About 1 in 5
available. But recently, some experts
Here’s how gene therapy works in the have suggested that hemophilia people with hemophilia A and
advanced clinical trial state: A modified gene therapy products could hit the 3 in 100 with hemophilia B will
virus is used to deliver a copy of the market soon. In 2020, two products develop inhibitors, or antibodies,
functional gene. According to a March looked close: BioMarin’s Roctavian in response to their treatments,
2021 article in the journal Hemasphere, for hemophilia A and uniQure’s according to the Centers for
recombinant adeno-associated viral AMT-061 for hemophilia B. But Disease Control and Prevention.
(AAV) vectors have recently been questions about both products Those with inhibitors cannot use
“the predominant transgene delivery interrupted their momentum. standard clotting factor treatment,
and they may not be eligible
for certain gene therapy products
Until gene therapy products are for some time, as very few

available for a wide group of people clinical trials are investigating
gene therapy in people affected
by inhibitors.
with bleeding disorders, many will still  Women with hemophilia. Most
need to prophylactically infuse—or women with hemophilia have mild

at least know how to infuse and have or moderate versions, while most
gene therapy clinical trials are
easy access to factor. investigating products for severe
hemophilia, which could leave
women out for a while. Access to
vehicle being used in hemophilia However, BioMarin recently gene therapy products for women
clinical gene therapy studies, an announced promising results from its will eventually depend on how the
approach thought to be largely Phase 3 gene therapy study in adults U.S. Food and Drug Administration
nonintegrating into the host genome.” with severe hemophilia A, and some (FDA) licenses or approves
are holding out hope that it might products, according to Clark.
Once the functional gene begins  Children with any severity of
to express the missing factor, get approval before too long. hemophilia A or B. Those under
the factor levels would begin to The question of “when” is still an 18 may not be eligible for gene
rise. Ideally, a person with severe unknown, though. As Joe Pugliese, therapy products for quite a while.
hemophilia would see their clotting president and CEO of Hemophilia
factor levels rise dramatically as the Alliance, said, “Gene therapy has been It’s also important to recognize that
result of undergoing some type of right around the corner for years.” a person who undergoes gene
gene therapy. No longer would they therapy will likely still need factor
have to worry as much about their Until gene therapy products are replacement. The gene therapy
factor levels and the possibility of available for a wide group of people product might only bring a person’s
spontaneous bleeds. with bleeding disorders, many will still clotting levels up to mild or even

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