Page 15 - HFA Dateline 2022 Special Edition
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still be able to transmit hemophilia to   risks over the long term,” Pipe said.   “Anything that can safely and
        their children,” Clark said.         “And the last overarching aspiration   effectively make a positive difference
                                             for gene therapy is we want to be     for quality of life is a good thing,”
        Some researchers are trying to       able to offer this to as many patients   she said. “Our job is to help people
        develop gene therapies that don’t use   as possible. The reality is none of the   understand what the risks and benefits
        viruses to deliver the gene, according   genetic therapies to date meet all   are. Some people are going to be early
        to Clark. “Some companies are using   the check boxes for our aspirations   adopters and some aren’t.”
        nanoparticles, which are small particles   yet. But there’s cause for optimism
        made of fats or lipids,” he said.                                          One critical consideration is that not
                                             that the current wave of therapies is
        Nanoparticle research is ongoing,    really going to offer something worth   everyone will be eligible for these
                                                                                   new treatments, Wilkes said. “Not
        Clark added. “Nanoparticles are      considering if they get approved by   everyone will meet the criteria to
        used now in mRNA vaccines for        the regulators.”                      have gene therapy,” she added.
        COVID-19,” he said, noting that      Sonji Wilkes, vice president of public
        using nanoparticles in gene therapy   affairs for HFA, shares that optimism   Among those who are ineligible are
        for bleeding disorders poses a       about gene therapy’s potential:       those younger than 18, according to
        particular challenge—getting the                                           Pipe. “We don’t do this method of
        factor-producing genes to the liver.
        “Adeno-associated viruses naturally
        target the liver,” Clark said. “But with   Emerging Gene Therapies
        nanoparticles, it’s going to be more of
                                                for People With
        a challenge to get them to the liver.”

        Optimism and Concern                    Bleeding Disorders
        Most people with bleeding disorders
        can effectively manage their            Several gene therapies are currently being tested in clinical trials in people
        condition with current treatments.      with moderately severe to severe hemophilia A and B. Data from some of
        So what might make gene therapy         these trials indicate that some gene therapies are showing promise.
        a better option for some? “We have
        well-established treatments and         In the 134 participants with hemophilia A who participated in a Phase 3
        protocols using factor-replacement      GENEr8-1 clinical trial of Roctavian (valoctocogene roxaparvovec), a gene
        therapy and now Hemlibra that are       therapy from BioMarin Pharmaceutical Inc., results showed that participants
        affording unprecedented bleed           maintained therapeutic levels of factor VIII two years after dosing.
        control and convenience for patients
        and allowing them to live their lives   Other gene therapies for hemophilia A currently in clinical trials include SPK-
        to potential,” Pipe said. “With that    8011 from Spark Therapeutics (a member of the Roche Group) and SB-525
        backdrop, what is gene therapy          from Pfizer Inc. in collaboration with Sangamo Therapeutics Inc.
        looking to achieve?”                    For 54 people with moderate to severe hemophilia B enrolled in the
                                                Phase 3 HOPE-B clinical trial, a single dose of etranacogene dezaparvovec,
        Pipe highlighted three achievements
        he hopes gene therapy will deliver:     a gene therapy from uniQure and CSL Behring that uses adeno-associated
        sustained full restoration of blood     virus 5 (AAV5), met its primary endpoint of reducing the annualized
        coagulation, a way to deliver long-     bleeding rate in participants when compared with factor IX prophylactic
        term effective treatment without the    therapy. The drug also increased endogenous factor IX activity and reduced
        need for frequent infusions, and an     the need for factor IX replacement therapy in participants.
        acceptable ratio of treatment safety    Another gene therapy for hemophilia B currently in a Phase 3 clinical trial is
        to effectiveness.                       fidanacogene elaparvovec from Pfizer Inc. and Spark Therapeutics.

        “Ideally, we want a genetic             To learn more about gene therapy clinical trials for people with bleeding
        therapy that is not going to have       disorders, visit
        unacceptable toxicities or significant

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