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    <title>Gene therapy</title>
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      <title>Columbia researchers use base editing to modify human embryo genome</title>
      <description>
        <![CDATA[Scientists at Columbia University have used base editing to make precise changes in the genomes of human embryos, avoiding the damage to chromosomes that occurs following two-stranded DNA cuts with conventional CRISPR/Cas9 editing.]]>
      </description>
      <guid>http://www.bioworld.com/articles/731851</guid>
      <pubDate>Wed, 10 Jun 2026 09:00:00 -0400</pubDate>
      <link>https://www.bioworld.com/articles/731851-columbia-researchers-use-base-editing-to-modify-human-embryo-genome</link>
      <media:content url="https://www.bioworld.com/ext/resources/BWS/BWS-library/DNA-gene-edit-genomics.webp?t=1747837759" type="image/jpeg" medium="image" fileSize="1061729">
        <media:title type="plain">DNA double helix under a magnifying glass</media:title>
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    </item>
    <item>
      <title>Sensorion selects SENS-601 as lead program for hearing loss</title>
      <description>
        <![CDATA[Sensorion SA has selected SENS-601 (GJB2-GT) as its lead program and has filed clinical trial applications to study its use for GJB2-related hearing loss. SENS-601 is an AAV-based gene therapy program, utilizing a gene therapy platform codeveloped with Institut Pasteur.]]>
      </description>
      <guid>http://www.bioworld.com/articles/731777</guid>
      <pubDate>Wed, 10 Jun 2026 09:00:00 -0400</pubDate>
      <link>https://www.bioworld.com/articles/731777-sensorion-selects-sens-601-as-lead-program-for-hearing-loss</link>
      <media:content url="https://www.bioworld.com/ext/resources/BWS/BWS-library/AAV-Adenovirus-DNA.webp?t=1747149016" type="image/jpeg" medium="image" fileSize="463671">
        <media:title type="plain">3D illustration showing DNA inside adenovirus</media:title>
      </media:content>
    </item>
    <item>
      <title>Columbia researchers use base editing to modify human embryo genome</title>
      <description>
        <![CDATA[Scientists at Columbia University have used base editing to make precise changes in the genomes of human embryos, avoiding the damage to chromosomes that occurs following two-stranded DNA cuts with conventional Crispr-Cas9 editing.]]>
      </description>
      <guid>http://www.bioworld.com/articles/731708</guid>
      <pubDate>Tue, 09 Jun 2026 12:00:00 -0400</pubDate>
      <link>https://www.bioworld.com/articles/731708-columbia-researchers-use-base-editing-to-modify-human-embryo-genome</link>
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        <media:title type="plain">DNA double helix under a magnifying glass</media:title>
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    </item>
    <item>
      <title>Idefine announces Kleefstra syndrome gene therapy collaboration</title>
      <description>
        <![CDATA[Idefine, The Kleefstra Syndrome Foundation, has established a collaboration with UT Southwestern Medical Center to advance development of a potential gene therapy to treat Kleefstra syndrome, a rare neurodevelopmental disorder caused by changes or loss of the <em>EHMT1</em> gene, which plays a critical role in brain development and function.]]>
      </description>
      <guid>http://www.bioworld.com/articles/731656</guid>
      <pubDate>Thu, 04 Jun 2026 09:00:00 -0400</pubDate>
      <link>https://www.bioworld.com/articles/731656-idefine-announces-kleefstra-syndrome-gene-therapy-collaboration</link>
      <media:content url="https://www.bioworld.com/ext/resources/Stock-images/Research-and-science/Gene-therapy-research-pic.webp?t=1588872515" type="image/png" medium="image" fileSize="467422">
        <media:title type="plain">DNA illustration</media:title>
      </media:content>
    </item>
    <item>
      <title>FDA advises leveraging what’s already known in gene therapy R&amp;D</title>
      <description>
        <![CDATA[Rather than reinventing the wheel for every gene therapy that uses genome editing, the U.S. FDA is advising sponsors on leveraging existing knowledge, be it publicly available or platform-based, to more efficiently advance their products across multiple stages of development.]]>
      </description>
      <guid>http://www.bioworld.com/articles/731529</guid>
      <pubDate>Tue, 02 Jun 2026 12:00:00 -0400</pubDate>
      <link>https://www.bioworld.com/articles/731529-fda-advises-leveraging-whats-already-known-in-gene-therapy-r-and-d</link>
      <media:content url="https://www.bioworld.com/ext/resources/BWS/BWS-library/DNA-gene-editing.webp?t=1730128296" type="image/jpeg" medium="image" fileSize="222415">
        <media:title type="plain">DNA double helix illustration with section being removed in red</media:title>
      </media:content>
    </item>
    <item>
      <title>Voyager’s tau-targeted gene therapy VY-1706 gains IND approval</title>
      <description>
        <![CDATA[Voyager Therapeutics Inc. has obtained IND clearance from the FDA for VY-1706, the company’s investigational gene therapy for the treatment of Alzheimer’s disease.]]>
      </description>
      <guid>http://www.bioworld.com/articles/731578</guid>
      <pubDate>Tue, 02 Jun 2026 09:00:00 -0400</pubDate>
      <link>https://www.bioworld.com/articles/731578-voyagers-tau-targeted-gene-therapy-vy-1706-gains-ind-approval</link>
      <media:content url="https://www.bioworld.com/ext/resources/Stock-images/Research-and-science/Science-tau-neuron.webp?t=1744640705" type="image/png" medium="image" fileSize="638411">
        <media:title type="plain">Tau neuron illustration</media:title>
        <media:description type="plain">Tau protein accumulating on neuron. Credit: Kenneth S. Kosik, University of California - Santa Barbara</media:description>
      </media:content>
    </item>
    <item>
      <title>ONYX-101 restores COL4A5 expression in X-linked Alport syndrome</title>
      <description>
        <![CDATA[X-linked Alport syndrome is an inherited kidney disease caused by pathogenic mutations in the <em>COL4A5</em> gene. Patients develop hematuria, proteinuria and kidney function decline leading to end-stage renal disease. Nionyx Bio Inc. has developed ONYX-101, a novel kidney-targeting therapeutic designed to ensure durable <em>COL4A5</em> restoration through dual-vector AAV delivery using NYX capsids that were optimized for kidney targeting.]]>
      </description>
      <guid>http://www.bioworld.com/articles/731296</guid>
      <pubDate>Wed, 27 May 2026 09:00:00 -0400</pubDate>
      <link>https://www.bioworld.com/articles/731296-onyx-101-restores-col4a5-expression-in-x-linked-alport-syndrome</link>
      <media:content url="https://www.bioworld.com/ext/resources/Stock-images/Therapeutic-topics/Nephrology/kidney-dna-gene-therapy.webp?t=1747406414" type="image/jpeg" medium="image" fileSize="710010">
        <media:title type="plain">Illustration of kidneys with DNA double helix</media:title>
      </media:content>
    </item>
    <item>
      <title>Circio and Aavigen to collaborate on targeted cardiac AAVs</title>
      <description>
        <![CDATA[Circio Holding ASA and Aavigen GmbH have entered a research collaboration focused on developing circVec-enhanced AAV vectors engineered for targeted, low-dose gene therapies for heart diseases.]]>
      </description>
      <guid>http://www.bioworld.com/articles/731292</guid>
      <pubDate>Wed, 27 May 2026 09:00:00 -0400</pubDate>
      <link>https://www.bioworld.com/articles/731292-circio-and-aavigen-to-collaborate-on-targeted-cardiac-aavs</link>
      <media:content url="https://www.bioworld.com/ext/resources/Stock-images/Therapeutic-topics/Cardiovascular/Heart-and-DNA.webp?t=1629916296" type="image/png" medium="image" fileSize="441817">
        <media:title type="plain">Heart and DNA</media:title>
      </media:content>
    </item>
    <item>
      <title>China’s new IIT rules expected to wipe out ‘gray market’ </title>
      <description>
        <![CDATA[New regulations tighten regulatory oversight of China’s investigator-initiated trials (IITs) but legitimize the pathway that will be open to other modalities beyond cell and gene therapies.]]>
      </description>
      <guid>http://www.bioworld.com/articles/731468</guid>
      <pubDate>Tue, 26 May 2026 11:00:00 -0400</pubDate>
      <link>https://www.bioworld.com/articles/731468-chinas-new-iit-rules-expected-to-wipe-out-gray-market</link>
      <media:content url="https://www.bioworld.com/ext/resources/Stock-images/Research-and-science/Stem-cells2.webp?t=1772820791" type="image/png" medium="image" fileSize="466079">
        <media:title type="plain">Stem cells </media:title>
      </media:content>
    </item>
    <item>
      <title>Gemma Biotherapeutics’ GB-703 shows promise for DMD</title>
      <description>
        <![CDATA[AAV-based therapies for Duchenne muscular dystrophy (DMD) have shown efficacy, but have limitations such as poor delivery to target tissues and toxicity associated with the vector. Gemma Biotherapeutics Inc. has developed a gene therapy candidate, GB-703, which uses a new myotropic, integrin-binding AAV capsid containing a codon-optimized, deimmunized hybrid payload.]]>
      </description>
      <guid>http://www.bioworld.com/articles/731275</guid>
      <pubDate>Tue, 26 May 2026 09:00:00 -0400</pubDate>
      <link>https://www.bioworld.com/articles/731275-gemma-biotherapeutics-gb-703-shows-promise-for-dmd</link>
      <media:content url="https://www.bioworld.com/ext/resources/BWS/BWS-library/DNA-wheel-chair-muscular-dystrophy.webp?t=1743173916" type="image/jpeg" medium="image" fileSize="91392">
        <media:title type="plain">Illustration of DNA double helix and motorized wheel chair</media:title>
      </media:content>
    </item>
    <item>
      <title>China’s new IIT rules expected to wipe out ‘gray market’ </title>
      <description>
        <![CDATA[New regulations tighten regulatory oversight of China’s investigator-initiated trials (IITs) but legitimize the pathway that will be open to other modalities beyond cell and gene therapies.]]>
      </description>
      <guid>http://www.bioworld.com/articles/731388</guid>
      <pubDate>Fri, 22 May 2026 12:00:00 -0400</pubDate>
      <link>https://www.bioworld.com/articles/731388-chinas-new-iit-rules-expected-to-wipe-out-gray-market</link>
      <media:content url="https://www.bioworld.com/ext/resources/Stock-images/Research-and-science/Stem-cells2.webp?t=1772820791" type="image/png" medium="image" fileSize="466079">
        <media:title type="plain">Stem cells </media:title>
      </media:content>
    </item>
    <item>
      <title>Asgard’s AT-108 shows promise as immunotherapeutic </title>
      <description>
        <![CDATA[Defects in antigen presentation lead to resistance to cancer immunotherapy, where type I conventional dendritic cells (cDC1s) are crucial drivers of antitumor immunity and their presence is tied to favorable responses and better outcomes. Intratumoral delivery of adenoviral vector, Ad5-PIB, encoding PU.1, IRF8 and BATF3 reprograms tumor cells into cDC1-like antigen-presenting cells and has shown synergy with immune checkpoint blockade (ICB) therapy at exerting antitumor immunity. Asgard Therapeutics AB has developed AT-108, a lead candidate developed for durable efficacy.]]>
      </description>
      <guid>http://www.bioworld.com/articles/731255</guid>
      <pubDate>Fri, 22 May 2026 09:00:00 -0400</pubDate>
      <link>https://www.bioworld.com/articles/731255-asgards-at-108-shows-promise-as-immunotherapeutic</link>
      <media:content url="https://www.bioworld.com/ext/resources/Stock-images/Research-and-science/Dendritic-cells.webp?t=1741357955" type="image/jpeg" medium="image" fileSize="413729">
        <media:title type="plain">Dendritic cells</media:title>
      </media:content>
    </item>
    <item>
      <title>Sangamo presents primate data for prion suppressor ST-506</title>
      <description>
        <![CDATA[Sangamo Therapeutics Inc. discussed gene regulation approaches for neurodegenerative diseases when presenting findings on their clinical candidate ST-506 for the treatment of prion disease. ]]>
      </description>
      <guid>http://www.bioworld.com/articles/731252</guid>
      <pubDate>Fri, 22 May 2026 09:00:00 -0400</pubDate>
      <link>https://www.bioworld.com/articles/731252-sangamo-presents-primate-data-for-prion-suppressor-st-506</link>
      <media:content url="https://www.bioworld.com/ext/resources/Stock-images/Therapeutic-topics/Neurology/brain-neuro-dna.webp?t=1745264981" type="image/jpeg" medium="image" fileSize="555555">
        <media:title type="plain">Brain and DNA</media:title>
      </media:content>
    </item>
    <item>
      <title>HSPCs delivering tissue-penetrating frataxin ameliorate Friedreich’s ataxia symptoms</title>
      <description>
        <![CDATA[Researchers at the University of London and collaborating institutions have developed a gene and cell therapy approach that enables sustained systemic frataxin protein delivery, improving motor performance and tissue pathology, and supporting a promising translational strategy for long-term disease stabilization in Friedreich’s ataxia patients.]]>
      </description>
      <guid>http://www.bioworld.com/articles/731178</guid>
      <pubDate>Wed, 20 May 2026 09:00:00 -0400</pubDate>
      <link>https://www.bioworld.com/articles/731178-hspcs-delivering-tissue-penetrating-frataxin-ameliorate-friedreichs-ataxia-symptoms</link>
      <media:content url="https://www.bioworld.com/ext/resources/Stock-images/Research-and-science/Stem-cells1.webp?t=1631910994" type="image/png" medium="image" fileSize="491784">
        <media:title type="plain">Stem cells</media:title>
      </media:content>
    </item>
    <item>
      <title>‘Detargeted’ targeted gene therapy improves activity in Pompe</title>
      <description>
        <![CDATA[A new strategy aims to improve gene therapy for Pompe disease by optimizing both the genetic component that restores the function of a deficient lysosomal enzyme and the vector that delivers it to the target tissue while avoiding the liver. The findings suggest that combining an optimized transgene with a targeted capsid could significantly enhance the effectiveness of gene therapy for Pompe disease.]]>
      </description>
      <guid>http://www.bioworld.com/articles/731174</guid>
      <pubDate>Wed, 20 May 2026 09:00:00 -0400</pubDate>
      <link>https://www.bioworld.com/articles/731174-detargeted-targeted-gene-therapy-improves-activity-in-pompe</link>
      <media:content url="https://www.bioworld.com/ext/resources/BWS/BWS-library/Acid-alpha-glucosidase-molecular-structure.webp?t=1779288468" type="image/jpeg" medium="image" fileSize="390572">
        <media:title type="plain">Acid alpha-glucosidase molecular structure isolated on black</media:title>
      </media:content>
    </item>
    <item>
      <title>GPX-002 restores β-cell function in a diabetes model </title>
      <description>
        <![CDATA[Type 2 diabetes is marked by insulin resistance coupled with insufficient insulin secretion due to early β-cell dysfunction and progressive loss of β-cell mass. Pdx1 and MafA, critical for maintaining β-cell function, are progressively reduced under metabolic stress and in patients, driving disease progression. Researchers at the University of Pittsburgh have reported efficacy data demonstrating successful pancreatic delivery of GPX-002, an AAV-Pdx1/MafA construct, in HFD mice.]]>
      </description>
      <guid>http://www.bioworld.com/articles/731140</guid>
      <pubDate>Mon, 18 May 2026 09:00:00 -0400</pubDate>
      <link>https://www.bioworld.com/articles/731140-gpx-002-restores-cell-function-in-a-diabetes-model</link>
      <media:content url="https://www.bioworld.com/ext/resources/BWS/BWS-library/Endocrine-metabolic-pancreas-anatomy-illustration.webp?t=1774020868" type="image/jpeg" medium="image" fileSize="655771">
        <media:title type="plain">Pancreas anatomy illustration</media:title>
      </media:content>
    </item>
    <item>
      <title>Launch of CGTxchange to reactivate cell and gene therapy programs</title>
      <description>
        <![CDATA[The American Society of Gene & Cell Therapy (ASGCT) and Orphan Therapeutics Accelerator (OTXL) have announced the public launch of CGTxchange, an AI-enhanced clearinghouse and marketplace built to help reactivate cell and gene therapy programs that have been shelved despite strong scientific and clinical evidence.]]>
      </description>
      <guid>http://www.bioworld.com/articles/731124</guid>
      <pubDate>Fri, 15 May 2026 09:00:00 -0400</pubDate>
      <link>https://www.bioworld.com/articles/731124-launch-of-cgtxchange-to-reactivate-cell-and-gene-therapy-programs</link>
      <media:content url="https://www.bioworld.com/ext/resources/Stock-images/Misc/Archive-room.webp?t=1778858165" type="image/jpeg" medium="image" fileSize="314054">
        <media:title type="plain">Dimly lit archive room filled with cardboard storage boxes</media:title>
      </media:content>
    </item>
    <item>
      <title>ASGCT 2026: Directed evolution in gene therapy</title>
      <description>
        <![CDATA[Directed evolution has become a central pillar in gene therapy. This engineering strategy enables the generation of more efficient variants of genetic editors and delivery vectors. Molecular diversification methods are increasingly sophisticated and are now accelerated by machine learning and AI tools, as showcased at the 29th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) held in Boston this week.]]>
      </description>
      <guid>http://www.bioworld.com/articles/731119</guid>
      <pubDate>Fri, 15 May 2026 09:00:00 -0400</pubDate>
      <link>https://www.bioworld.com/articles/731119-asgct-2026-directed-evolution-in-gene-therapy</link>
      <media:content url="https://www.bioworld.com/ext/resources/BWS/BWS-library/DNA-and-genome-editing.webp?t=1778858165" type="image/jpeg" medium="image" fileSize="96216">
        <media:title type="plain">DNA and genome editing illustration</media:title>
      </media:content>
    </item>
    <item>
      <title>Will FDA show Affinity for fast yes to Regenxbio in DMD?</title>
      <description>
        <![CDATA[How the U.S. FDA might view the latest Duchenne muscular dystrophy (DMD) phase III data with gene therapy RGX-202 became the question for Regenxbio Inc., shares of which &nbsp;(NASDAQ:RGNX) closed May 14 at $6.25, down 38%, or $3.80, after the results were made public.]]>
      </description>
      <guid>http://www.bioworld.com/articles/731196</guid>
      <pubDate>Thu, 14 May 2026 12:00:00 -0400</pubDate>
      <link>https://www.bioworld.com/articles/731196-will-fda-show-affinity-for-fast-yes-to-regenxbio-in-dmd</link>
      <media:content url="https://www.bioworld.com/ext/resources/2023/06/02/Green-arrow-up-red-arrow-down.webp?t=1685735888" type="image/jpeg" medium="image" fileSize="43614">
        <media:title type="plain">Green arrow up red arrow down</media:title>
      </media:content>
    </item>
    <item>
      <title>KHN-921 gains IND clearance for &lt;em&gt;MYBPC3&lt;/em&gt;-associated HCM</title>
      <description>
        <![CDATA[Chengdu Origen Biotechnology Co. Ltd. and Vanotech Ltd. have announced IND clearance by the FDA for KHN-921 for the treatment of hypertrophic cardiomyopathy (HCM) associated with <em>MYBPC3</em> mutations.]]>
      </description>
      <guid>http://www.bioworld.com/articles/731095</guid>
      <pubDate>Thu, 14 May 2026 09:00:00 -0400</pubDate>
      <link>https://www.bioworld.com/articles/731095-khn-921-gains-ind-clearance-for-emmybpc3-em-associated-hcm</link>
      <media:content url="https://www.bioworld.com/ext/resources/Stock-images/Therapeutic-topics/Cardiovascular/Abstract-blue-human-heart-with-red-cardio-pulse-line-and-red-circle.webp?t=1752266610" type="image/png" medium="image" fileSize="259569">
        <media:title type="plain">Abstract blue human heart with red cardio pulse line and red circle</media:title>
      </media:content>
    </item>
    <item>
      <title>ASGCT 2026: Uncovering the mechanisms of AAV toxicity</title>
      <description>
        <![CDATA[Gene therapies rely on vectors to reach the target tissue where they act, such as adeno-associated viruses (AAVs) or lipid nanoparticles (LNPs), among other delivery strategies. Each combination is optimized for a specific cell type and indication, aiming to overcome challenges such as efficacy, specificity and toxicity. On May 13, 2026, two sessions included in the scientific symposia of the 29th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), being held in Boston this week, addressed AAV-related toxicities, which have led to fatal cases in clinical trials and remain an area for improvement in approved therapies.]]>
      </description>
      <guid>http://www.bioworld.com/articles/731091</guid>
      <pubDate>Thu, 14 May 2026 09:00:00 -0400</pubDate>
      <link>https://www.bioworld.com/articles/731091-asgct-2026-uncovering-the-mechanisms-of-aav-toxicity</link>
      <media:content url="https://www.bioworld.com/ext/resources/BWS/BWS-library/Colorful-adeno-associated-viruses-AAVs.webp?t=1778776816" type="image/png" medium="image" fileSize="1832336">
        <media:title type="plain">3D illustration of adeno-associated viruses</media:title>
      </media:content>
    </item>
    <item>
      <title>ASGCT 2026: Circular RNA, the new beast in gene and cell therapy</title>
      <description>
        <![CDATA[Circular RNA (circRNA) is not a new concept, but it is a novel strategy in the field of gene and cell therapy. While mRNA vaccines have revolutionized medicine, this RNA fragment without free ends surpasses their performance in both efficacy and durability, bringing it to the attention of several pioneering companies. The latest advances in circRNA presented at the 29th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) clearly surpass the performance achieved with linear mRNA.]]>
      </description>
      <guid>http://www.bioworld.com/articles/731268</guid>
      <pubDate>Wed, 13 May 2026 09:00:00 -0400</pubDate>
      <link>https://www.bioworld.com/articles/731268-asgct-2026-circular-rna-the-new-beast-in-gene-and-cell-therapy</link>
      <media:content url="https://www.bioworld.com/ext/resources/BWS/BWS-library/glowing-circle-illustration.webp?t=1778686093" type="image/png" medium="image" fileSize="673869">
        <media:title type="plain">Illustration of a glowing circle to represent circRNA</media:title>
      </media:content>
    </item>
    <item>
      <title>BTX-001 enables single-dose treatment for geographic atrophy</title>
      <description>
        <![CDATA[Researchers from Beacon Therapeutics Holdings Ltd. reported the development of BTX-001, an intravitreal gene therapy designed to target the complement pathway through delivery of a C5 inhibitor.]]>
      </description>
      <guid>http://www.bioworld.com/articles/731075</guid>
      <pubDate>Wed, 13 May 2026 09:00:00 -0400</pubDate>
      <link>https://www.bioworld.com/articles/731075-btx-001-enables-single-dose-treatment-for-geographic-atrophy</link>
      <media:content url="https://www.bioworld.com/ext/resources/Stock-images/Therapeutic-topics/Ocular/Eye-and-DNA.webp?t=1621374169" type="image/png" medium="image" fileSize="619372">
        <media:title type="plain">Eye and DNA</media:title>
      </media:content>
    </item>
    <item>
      <title>MHRA sets out proposal to redefine gene therapies</title>
      <description>
        <![CDATA[The U.K. Medicines and Healthcare products Regulatory Agency (MHRA) has opened a consultation on changes it is proposing to the legal definition of gene therapies, to reflect the advances in technology and manufacturing over the two decades since the current legislation was drawn up. The aim is to correct the mismatch between regulatory terminology and modern science, which MHRA says “can lead to uncertainty” over how a product is classified and to “inconsistent oversight and potential barriers for developers."]]>
      </description>
      <guid>http://www.bioworld.com/articles/731050</guid>
      <pubDate>Tue, 12 May 2026 12:00:00 -0400</pubDate>
      <link>https://www.bioworld.com/articles/731050-mhra-sets-out-proposal-to-redefine-gene-therapies</link>
      <media:content url="https://www.bioworld.com/ext/resources/Stock-images/Research-and-science/thumb/DNA-sequencing1.webp?t=1573772611" type="image/png" medium="image" fileSize="469891">
        <media:title type="plain">DNA-sequencing.png</media:title>
      </media:content>
    </item>
    <item>
      <title>Fractyl Health’s RJVA-001 cleared to enter clinic in Netherlands</title>
      <description>
        <![CDATA[Fractyl Health Inc. has received clinical trial application authorization in the Netherlands to initiate a first-in-human phase I/II study of RJVA-001, the first clinical candidate from the company’s Rejuva Smart GLP-1 gene therapy platform. ]]>
      </description>
      <guid>http://www.bioworld.com/articles/731012</guid>
      <pubDate>Tue, 12 May 2026 09:00:00 -0400</pubDate>
      <link>https://www.bioworld.com/articles/731012-fractyl-healths-rjva-001-cleared-to-enter-clinic-in-netherlands</link>
      <media:content url="https://www.bioworld.com/ext/resources/Stock-images/Research-and-science/Beta-cells.webp?t=1778592394" type="image/png" medium="image" fileSize="224899">
        <media:title type="plain">Beta cells</media:title>
        <media:description type="plain">β cell. Credit: Melton laboratory, Harvard University</media:description>
      </media:content>
    </item>
    <item>
      <title>Alzheimer’s, beyond the brain</title>
      <description>
        <![CDATA[Researchers at Daping Hospital in China have reported that liver-targeted delivery of the APOE3-Christchurch (APOE3Ch) variant, a rare protective form of apolipoprotein E, can indirectly reduce brain pathology, highlighting the therapeutic potential of peripheral approaches to Alzheimer’s disease.]]>
      </description>
      <guid>http://www.bioworld.com/articles/730993</guid>
      <pubDate>Mon, 11 May 2026 09:00:00 -0400</pubDate>
      <link>https://www.bioworld.com/articles/730993-alzheimers-beyond-the-brain</link>
      <media:content url="https://www.bioworld.com/ext/resources/Stock-images/Therapeutic-topics/Neurology/Brain-maze.webp?t=1636060283" type="image/png" medium="image" fileSize="122799">
        <media:title type="plain">Brain maze</media:title>
      </media:content>
    </item>
    <item>
      <title>Entrada sinks on mixed DMD data with ENTR-601-44</title>
      <description>
        <![CDATA[A lower-than-expected increase in dystrophin over baseline in the first and lowest-dose cohort of a phase I/II study of ENTR-601-44 in Duchenne muscular dystrophy (DMD) caused shares of Entrada Therapeutics Inc. to plunge more than 57%, despite the cohort meeting the safety and tolerability primary objective.]]>
      </description>
      <guid>http://www.bioworld.com/articles/730967</guid>
      <pubDate>Thu, 07 May 2026 12:00:00 -0400</pubDate>
      <link>https://www.bioworld.com/articles/730967-entrada-sinks-on-mixed-dmd-data-with-entr-601-44</link>
      <media:content url="https://www.bioworld.com/ext/resources/Stock-images/Money/Red-arrow-pointing-downward-on-a-stock-market-ticker.webp?t=1746653867" type="image/jpeg" medium="image" fileSize="239034">
        <media:title type="plain">Red arrow pointing downward on a stock market ticker</media:title>
      </media:content>
    </item>
    <item>
      <title>Apertura Gene Therapy and TSC Alliance partner on gene therapies</title>
      <description>
        <![CDATA[Apertura Gene Therapy LLC and the TSC Alliance have established a collaboration to advance gene therapy programs designed to treat tuberous sclerosis complex (TSC).]]>
      </description>
      <guid>http://www.bioworld.com/articles/730908</guid>
      <pubDate>Wed, 06 May 2026 09:00:00 -0400</pubDate>
      <link>https://www.bioworld.com/articles/730908-apertura-gene-therapy-and-tsc-alliance-partner-on-gene-therapies</link>
      <media:content url="https://www.bioworld.com/ext/resources/Stock-images/Deals-and-MAs/Deal-illustration1.webp?t=1613684293" type="image/png" medium="image" fileSize="542426">
        <media:title type="plain">Handshake with DNA, molecules</media:title>
      </media:content>
    </item>
    <item>
      <title>Elaaj Bio advances gene therapy for CDKL5 deﬁciency disorder</title>
      <description>
        <![CDATA[Elaaj Bio, a wholly owned subsidiary of the nonproﬁt Loulou Foundation, has entered into a partnership with Viralgen Vector Core SL to advance a gene therapy program for CDKL5 deﬁciency disorder.]]>
      </description>
      <guid>http://www.bioworld.com/articles/730904</guid>
      <pubDate>Wed, 06 May 2026 09:00:00 -0400</pubDate>
      <link>https://www.bioworld.com/articles/730904-elaaj-bio-advances-gene-therapy-for-cdkl5-deciency-disorder</link>
      <media:content url="https://www.bioworld.com/ext/resources/BWS/BWS-library/Brain-DNA-Pills.webp?t=1683902288" type="image/jpeg" medium="image" fileSize="47499">
        <media:title type="plain">Concept art for gene-therapy treatment for brain</media:title>
      </media:content>
    </item>
    <item>
      <title>AAV9-delivered AntagoNATs have preclinical efficacy as one-time treatment for Dravet syndrome</title>
      <description>
        <![CDATA[Dravet syndrome is a rare, severe, lifelong developmental and epileptic encephalopathy that begins in infancy and is marked by prolonged, often fever-triggered seizures that are difficult to control. It is usually caused by mutations in the SCN1A gene and is associated with developmental delay, cognitive and behavioral impairment, and reduced life expectancy.]]>
      </description>
      <guid>http://www.bioworld.com/articles/730814</guid>
      <pubDate>Mon, 04 May 2026 09:00:00 -0400</pubDate>
      <link>https://www.bioworld.com/articles/730814-aav9-delivered-antagonats-have-preclinical-efficacy-as-one-time-treatment-for-dravet-syndrome</link>
      <media:content url="https://www.bioworld.com/ext/resources/Stock-images/Therapeutic-topics/Neurology/Brain-genetics.webp?t=1589293930" type="image/png" medium="image" fileSize="402105">
        <media:title type="plain">Brain-DNA illustration</media:title>
      </media:content>
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