gene therapy

Gamida Cell Presents New Data from NAM-Enabled Genetically Modified Natural Killer (NK) Pipeline at International Society for Cell & Gene Therapy 2022


Poster selected for inclusion in conference’s Elevator Pitch Session: GDA-301 produces enhanced potency and persistence with combined genetic manipulation of CISH gene editing and the engineered expression of membrane-bound IL-15 for targeting hematologic malignancies and solid tumors GDA-601 generates promising immunotherapeutic potential to target multiple myeloma cells Company plans to select a genetically modified NK…

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PacBio transforms access to the epigenome and streamlines workflows


Significant enhancements to the Sequel II/IIe platform include methylation calling in native DNA, greatly accelerated sample preparation, and support for gene therapy applications Excerpt from the Press Release: MENLO PARK, Calif., April 21, 2022 (GLOBE NEWSWIRE) — PacBio (NASDAQ: PACB), a leading developer of high-quality, highly accurate sequencing solutions, today announced the release of a transformative capability…

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Ocugen, Inc. Announces First Patient Dosed in Phase 1/2 Clinical Trial for Gene Therapy Candidate OCU400 to Treat Inherited Retinal Degeneration


Announcement marks first clinical trial in humans of Ocugen’s modifier gene therapy platform Excerpt from the Press Release: MALVERN, Pa., April 01, 2022 (GLOBE NEWSWIRE) — Ocugen, Inc. (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene therapies, biologicals and vaccines, announced that the first patient has been dosed in the Phase 1/2…

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Thermo Fisher Scientific to Support National Program to Accelerate SARS-CoV-2 Variant Identification


Excerpt from the Press Release: CARLSBAD, Calif.–(BUSINESS WIRE)–Thermo Fisher Scientific Inc., the world leader in serving science, today announced its collaboration with the National Institutes of Health (NIH) Rapid Acceleration of Diagnostics (RADx) Initiative, Helix, and Rosalind aimed at developing a new genotyping method for SARS-CoV-2 that could speed up the identification of variants as…

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Passage Bio Doses First Patient in Global Clinical Trial for Infantile Krabbe Disease, A Rare Fatal Pediatric Condition


Company expects to report interim safety and biomarker data for Cohort 1 patients in the global phase 1/ 2 clinical trial, GALax-C, by end of 2022 Excerpt from the Press Release: PHILADELPHIA, March 10, 2022 (GLOBE NEWSWIRE) — Passage Bio, Inc. (Nasdaq: PASG), a clinical-stage genetic medicines company focused on developing transformative therapies for central…

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Epic Sciences scales its workflow for ctDNA testing with Next Generation Sequencing Leader Fulgent Fulgent Genetics’ expertise in high-volume testing powers Epic’s Comprehensive Cancer Profiling Excerpt from the Press Release: SAN DIEGO, Feb. 24, 2022 /PRNewswire/ –Epic Sciences, Inc. today announced it has partnered with Fulgent Genetics (NASDAQ:FLGT) to deliver DefineMBC comprehensive profiling results for metastatic breast…

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Myrtelle Announces Positive Preliminary Clinical Data for Its Proprietary Gene Therapy in Canavan Disease


Myrtelle’s Phase 1/2 clinical trial introduces first-of-its-kind proprietary recombinant adeno-associated virus (rAAV) vector designed to enable targeting of oligodendrocytes To date, 3 patients received gene therapy with the rAAV vector at a dose of 3.7 x 1013 vg delivered via intracerebroventricular administration Available follow-up data in treated patients demonstrate favorable safety and tolerability with encouraging initial…

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Kriya Licenses Next Generation Complement-Targeted Gene Therapies for the Treatment of Geographic Atrophy and Other Ocular Diseases


Excerpt from the Press Release: REDWOOD CITY, Calif. & RESEARCH TRIANGLE PARK, N.C.–(BUSINESS WIRE)–Kriya Therapeutics, Inc., a fully integrated company pioneering novel technologies and therapeutics in gene therapy, today announced an exclusive agreement with the Medical University of South Carolina (MUSC) Foundation for Research Development to license next generation complement-targeted gene therapies for the treatment…

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BridgeBio Pharma Announces Dosing of First Patient in Phase 1/2 Trial of Investigational Gene Therapy for Congenital Adrenal Hyperplasia (CAH)


– If successful, BridgeBio’s investigational gene therapy BBP-631 would be the first therapy for CAH to restore the body’s hormone and steroid balance by enabling people with CAH to make their own cortisol and aldosterone -Initial Phase 1/2 data readout anticipated in the second half of 2022 -BridgeBio’s gene therapy portfolio also includes a clinical stage…

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4D Molecular Therapeutics Announces First Patient Dosed in Phase 1/2 Clinical Trial of 4D-150, a Dual-Transgene Intravitreal Gene Therapy for Patients with wet AMD


Excerpt from the Press Release: EMERYVILLE, Calif., Jan. 06, 2022 (GLOBE NEWSWIRE) — 4D Molecular Therapeutics, Inc. (4DMT) (Nasdaq: FDMT), a clinical-stage gene therapy company harnessing the power of directed evolution for targeted gene therapies, announced that the first patient has been dosed in the Phase 1/2 clinical trial of 4D-150 for neovascular age-related macular…

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