AVLAYAH and KRESLADI Win FDA Accelerated Approvals

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1.Denali Therapeutics. Denali Therapeutics announces U.S. FDA approval of AVLAYAH™ (tividenofusp alfa-eknm) for treatment of hunter syndrome (MPS II). 2026. Available from: https://investors.denalitherapeutics.com/news-releases/news-release-details/denali-therapeutics-announces-us-fda-approval-avlayahtm.

2.Rocket Pharmaceuticals. Rocket pharmaceuticals announces FDA approval of KRESLADI™ for pediatric patients with severe Leukocyte Adhesion Deficiency-I (LAD-I). 2026. Available from: https://ir.rocketpharma.com/news-releases/news-release-details/rocket-pharmaceuticals-announces-fda-approval-kresladitm [Last accessed: April 1, 2026].

3.Sangamo Therapeutics. Sangamo therapeutics reports recent business highlights and fourth quarter and full year 2025 financial results. 2026. Available from: https://investor.sangamo.com/news-releases/news-release-details/sangamo-therapeutics-reports-recent-business-highlights-and-15 [Last accessed: April 1, 2026].

4.Ultragenyx Pharmaceutical. Ultragenyx announces U.S. FDA acceptance of BLA resubmission for UX111 AAV gene therapy to treat Sanfilippo syndrome type A (MPS IIIA). 2026. Available from: https://ir.ultragenyx.com/news-releases/news-release-details/ultragenyx-announces-us-fda-acceptance-bla-resubmission-ux111 [Last accessed: April 3, 2026].

5.Cytiva and San Raffaele Telethon Institute for Gene Therapy (SR-TIGET). Cytiva and San Raffaele Telethon Institute for Gene Therapy (SR-TIGET) launch collaboration to advance next-generation genomic medicine platforms. 2026. Available from: https://www.fondazionetelethon.it/en/stories-and-news/news/from-telethon-foundation/cytiva-and-san-raffaele-telethon-institute-for-gene-therapy-sr-tiget-launch-collaboration-to-advance-next-generation-genomic-medicine-platforms/ [Last accessed: April 1, 2026].

6.Ray Therapeutics. Ray therapeutics receives FDA Regenerative Medicine Advanced Therapy (RMAT) designation for RTx-015 in retinitis Pigmentosa. 2026. Available from: https://www.businesswire.com/news/home/20260401189848/en/Ray-Therapeutics-Receives-FDA-Regenerative-Medicine-Advanced-Therapy-RMAT-Designation-for-RTx-015-in-Retinitis-Pigmentosa [Last accessed: April 1, 2026].

7.Ocugen. Ocugen announces topline 12-month data from phase 2 ArMaDa clinical trial evaluating OCU410 modifier gene therapy for geographic atrophy secondary to dry age-related macular degeneration. 2026. Available from: https://ir.ocugen.com/news-releases/news-release-details/ocugen-announces-topline-12-month-data-phase-2-armada-clinical [Last accessed: April 1, 2026].

8.Apertura Gene Therapy and Viralgen. Apertura gene therapy and viralgen announce strategic collaboration to manufacture TfR1 CapX, a next-generation capsid for gene therapies designed to treat central nervous system diseases. 2026. Available from: https://www.prnewswire.com/news-releases/apertura-gene-therapy-and-viralgen-announce-strategic-collaboration-to-manufacture-tfr1-capx-a-next-generation-capsid-for-gene-therapies-designed-to-treat-central-nervous-system-diseases-302714462.html [Last accessed: April 5, 2026].