DNA RNA and Cells

07 Jan 2022 Freeline Announces FDA Clearance of Investigational New Drug Application for FLT201 for Gaucher Disease Type 1
07 Jan 2022 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
07 Jan 2022 REGENXBIO Announces FDA Clearance of IND for Clinical Trial of RGX-202, a Novel Gene Therapy Candidate for Duchenne Muscular Dystrophy
07 Jan 2022 Cellusion Raises 1.1B JPY to Advance Japan and Global Clinical Trials of CLS001 for Corneal Endothelial Cell Regenerative Therapy
06 Jan 2022 BlueRock Therapeutics Announces Closeout of First Cohort in Phase 1 Trial in Patients with Advanced Parkinson's Disease
05 Jan 2022 Dystrogen Therapeutics Announces First in Man Dosing of Novel Chimeric Cell Therapy for Duchenne Muscular Dystrophy and Reports 6-week Clinical Outcomes
05 Jan 2022 AVROBIO Reprioritizes Pipeline Programs
04 Jan 2022 Belief BioMed Completed Dosing of the First Subject in the Registrational Gene Therapy Clinical Trial for Hemophilia B
29 Dec 2021 RIBOMIC Announces Preliminary Topline Data From Phase 2 Trials of RBM-007 for Wet Age-Related Macular Degeneration
23 Dec 2021 FDA approves Novartis Leqvio® (inclisiran), first-in-class siRNA to lower cholesterol and keep it low with two doses a year
21 Dec 2021 Sorrento Therapeutics Announces FDA Clearance of Its IND Application of Next Generation oHSV Oncolytic Virus STI-1386 for Patients With Relapsed and Refractory Solid Tumors
21 Dec 2021 Alnylam Submits CTA Application for ALN-XDH, an Investigational RNAi Therapeutic for the Treatment of Gout
21 Dec 2021 bluebird bio Announces Partial Clinical Hold for Patients Under 18 in Sickle Cell Gene Therapy Clinical Program
21 Dec 2021 Alnylam Initiates Phase 2 Study of Lumasiran in Patients with Recurrent Kidney Stone Disease
20 Dec 2021 Editas Medicine Announces FDA Clearance of Investigational New Drug (IND) Application for EDIT-301 for the Treatment of Transfusion-Dependent Beta Thalassemia
20 Dec 2021 Lineage Establishes Exclusive Worldwide Collaboration With Genentech for the Development and Commercialization of OpRegen® RPE Cell Therapy for the Treatment of Ocular Disorders
18 Dec 2021 bluebird bio Announces FDA Priority Review of Biologics License Application for eli-cel Gene Therapy for Cerebral Adrenoleukodystrophy (CALD) in Patients Without a Matched Sibling Donor
17 Dec 2021 ProQR Announces First Patients Dosed in Phase 2/3 Pivotal Trials of QR-421a for USH2A Mediated Retinitis Pigmentosa
17 Dec 2021 IN8bio Provides Update from the Ongoing Phase 1 Clinical Trial of its Allogeneic Gamma-Delta T Cell Therapy in Leukemia Patients Undergoing Hematopoietic Stem Cell Transplant
17 Dec 2021 uniQure Announces Clinical Update on First Patients in Phase I/II Clinical Trial of AMT-130 Gene Therapy for the Treatment of Huntington’s Disease
17 Dec 2021 Lyell Immunopharma Announces FDA Clearance of its IND for LYL797, a CAR T-Cell Therapy Incorporating Novel Reprogramming Technologies for Solid Tumors
17 Dec 2021 BioMarin and Skyline Therapeutics Announce Strategic Collaboration to Develop Novel Gene Therapies for Cardiovascular Diseases
17 Dec 2021 Taysha Gene Therapies Announces Initiation of Clinical Development of TSHA-118 for the Treatment of CLN1 Disease
16 Dec 2021 EMA Grants Accelerated Assessment for CSL Behring's Haemophilia B Gene Therapy
16 Dec 2021 invIOs goes live with unique cell therapy platform and clinical-stage immuno-oncology pipeline

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