DNA RNA and Cells

21 Dec 2022 Alnylam Submits CTA Application for ALN-KHK, an Investigational RNAi Therapeutic for the Treatment of Type 2 Diabetes
21 Dec 2022 Orgenesis and Kurve Therapeutics Announce Pre-Clinical Study Results for a Cell-Based Oncolytic Virus Bearing Product Demonstrating Over 50% Reduction in a Murine Glioblastoma Model
21 Dec 2022 Magenta Therapeutics Provides Update for MGTA-117 Phase 1/2 Dose Escalation Clinical Trial
21 Dec 2022 Sirnaomics Announces Interim Data from Phase I Clinical Trial of RNAi Therapeutic STP707 for Treatment of Multiple Solid Tumors
20 Dec 2022 Entrada Therapeutics Announces Clinical Hold on IND Application for ENTR-601-44 in Duchenne Muscular Dystrophy
20 Dec 2022 Atara Biotherapeutics' Ebvallo® (tabelecleucel) Receives European Commission Approval as First Ever Therapy for Adults and Children with EBV+ PTLD
20 Dec 2022 Neurophth Receives IND Clearance from FDA for AAV-ND1 Gene Therapy of LHON
17 Dec 2022 Ferring Receives Approval from U.S. FDA for Adstiladrin for High-Risk, BCG-Unresponsive Non-Muscle Invasive Bladder Cancer
15 Dec 2022 Nexcella, Inc. in-licenses NXC-201, BCMA-targeted Next-Generation CAR-T Therapy Demonstrating High Complete Response Rate in Heavily Pre-Treated Multiple Myeloma (71% Complete Responses) and AL Amyloidosis (100% Complete Responses)
15 Dec 2022 Aligos Therapeutics Initiates Dosing with its Small Interfering RNA (siRNA) Drug Candidate, ALG-125755, in Subjects with Chronic Hepatitis B (CHB)
15 Dec 2022 SpliSense Initiates Phase 1/2 Study of SPL84, RNA-Based Therapy, for the Treatment of Cystic Fibrosis
15 Dec 2022 Avidity Announces Positive AOC 1001 Phase 1/2 MARINA™ Data Demonstrating First-Ever Successful Targeted Delivery of RNA to Muscle - Revolutionary Advancement for the Field of RNA Therapeutics (PRNewsfoto/Avidity Biosciences, Inc.)
15 Dec 2022 Seelos Therapeutics Announces Data Demonstrating Downregulation of Alpha Synuclein in an In Vivo Gene Therapy Study of SLS-004 Utilizing CRISPR-dCas9 in Parkinson's Disease
15 Dec 2022 SwanBio Therapeutics Initiates First-in-Human Study of AAV Gene Therapy for Adrenomyeloneuropathy
14 Dec 2022 Exicure, Inc. Announces Termination of AbbVie and Ipsen Collaboration Agreements
13 Dec 2022 Elixirgen Therapeutics Presents New Clinical Data Demonstrating Ex Vivo Telomere Elongation with EXG-34217 in Dyskeratosis Congenita at the 64th ASH Annual Meeting
13 Dec 2022 Precigen Announces Positive Phase 1 Dose Escalation Data for Autologous PRGN-3006 UltraCAR-T® Manufactured Overnight for Next Day Infusion in Relapsed or Refractory Acute Myeloid Leukemia Patients
13 Dec 2022 AvenCell Presents Safety and Efficacy Data from Lead CD123-Directed Universal CAR T Cell Therapy Program at ASH 2022 Meeting
12 Dec 2022 Rocket Pharmaceuticals Presents Positive Clinical Data from Fanconi Anemia, Pyruvate Kinase Deficiency and Severe Leukocyte Adhesion Deficiency-I Programs at the 64th American Society of Hematology (ASH) Annual Meeting
12 Dec 2022 Orchard Therapeutics Announces Promising Early Neurocognitive Outcomes from Ongoing Proof-of-concept Study of OTL-201 in MPS-IIIA
12 Dec 2022 Vertex Announces Investigational New Drug (IND) Application for VX-522, mRNA Therapy for People With Cystic Fibrosis, Cleared by FDA
11 Dec 2022 Fate Therapeutics Announces Clinical Safety and Activity Data of First-ever iPSC-derived CAR T-cell Therapy at 2022 ASH Annual Meeting
11 Dec 2022 Poseida Therapeutics Presents Preclinical Data from P-FVIII-101 Gene Therapy for Hemophilia A at the 64th ASH Annual Meeting & Exposition
11 Dec 2022 Wugen Presents Data Supporting Clinical Development of WU-NK-101 for Relapsed / Refractory AML at the American Society of Hematology’s (ASH) 64th Annual Meeting
11 Dec 2022 Gracell Biotechnologies Presents Clinical Data for FasTCAR-T GC012F for High Risk, Newly Diagnosed Multiple Myeloma Demonstrating 100% Overall Response Rate

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