DNA RNA and Cells

31 Mar 2019 Atara Biotherapeutics Announces Collaborator Presentation of Positive Phase 1 Clinical Results for a Mesothelin-Targeted CAR T Immunotherapy in Patients with Advanced Mesothelioma
30 Mar 2019 Dicerna™ Announces the Presentation of Updated Data Demonstrating Utility of its Lead Compound DCR-PHXC in Treating Primary Hyperoxaluria Type 1 (PH1) and Type 2 (PH2)
30 Mar 2019 bluebird bio Receives Positive Opinion from CHMP for ZYNTEGLO™ (autologous CD34+ cells encoding βA-T87Q-globin gene) Gene Therapy for Patients 12 Years and Older with Transfusion-Dependent β-Thalassemia (TDT) Who Do Not Have β0/β0 Genotype
30 Mar 2019 Celularity Announces Results of Phase 1 Studies of PNK-007, an Allogeneic, Off-the-Shelf, Placental-Derived Cell Therapy, at AACR Annual Meeting 2019
26 Mar 2019 Autolus Therapeutics Unveils Expanded Next Generation Technology and Pipeline and Provides Key Update on AUTO3 Program in Pediatric ALL at R&D Day
26 Mar 2019 Oxford Biomedica notes the Japanese approval of Kymriah® (tisagenlecleucel), the first CAR-T cell therapy authorised in Asia
21 Mar 2019 MolMed received the authorization to start phase I/II clinical investigation with CAR T CD44v6 cells in acute myeloid leukemia and multiple myeloma.
21 Mar 2019 Adlai Nortye Receives NMPA Approval for Pelareorep (AN1004) in China to Initiate Phase III Clinical Trials
20 Mar 2019 Trizell Ltd. announces Phase 3 pivotal study of interferon alfa-2b gene therapy in malignant pleural mesothelioma
19 Mar 2019 Arrowhead Pharmaceuticals Files IND for Pivotal Phase 2/3 Study of ARO-AAT for Treatment of Alpha-1 Liver Disease
14 Mar 2019 BrainStorm Announces First Patient Enrolled in Phase 2 Clinical Trial of Progressive MS
13 Mar 2019 PsiOxus Therapeutics Announces First-in-Human Dosing of their Second Gene Therapy Cancer Treatment
11 Mar 2019 ProQR Doses First Patient in Phase 1/2 STELLAR Trial of QR-421a for Usher Syndrome Type 2
11 Mar 2019 Rubius Therapeutics Announces FDA Clearance of Investigational New Drug Application for First-Ever Red Cell Therapeutic, RTX-134, for Treatment of Phenylketonuria
11 Mar 2019 Alnylam Submits CTA Application for ALN-AGT, an Investigational RNAi Therapeutic for the Treatment of Hypertension in High Unmet Need Settings
11 Mar 2019 Arrowhead Pharmaceuticals Begins Dosing in Phase 1 Study of ARO-APOC3 for Treatment of Hypertriglyceridemia
07 Mar 2019 AOBiome Doses First Patient in Phase 1b Clinical Trial of B244 for the Treatment of Pediatric Atopic Dermatitis
07 Mar 2019 Alnylam Reports Positive Topline Results from ENVISION Phase 3 Study of Givosiran in Acute Hepatic Porphyria
06 Mar 2019 NKMax America Announces FDA Approval of IND for Phase 1 Trial of SNK01 in Patients with Refractory Cancer
02 Mar 2019 CARsgen Therapeutics Receives IND Clearance for BCMA-CAR-T Cells from the NMPA
02 Mar 2019 Akcea and Ionis Receive Positive EU CHMP Opinion for WAYLIVRATM (volanesorsen)
28 Feb 2019 Translate Bio Provides Updates on Cystic Fibrosis (CF) and Ornithine Transcarbamylase (OTC) Deficiency Programs
27 Feb 2019 Esperite N.V. (ESP) with The Cell Factory has confirmed a long-term effect of extracellular vesicle drug candidate CF-MEV-132 for the treatment of Bronchopulmonary Dysplasia
27 Feb 2019 Idera Pharmaceuticals Completes Enrollment Into the ILLUMINATE-204 Trial of the Combination of Tilsotolimod and Ipilimumab for Unresectable or Metastatic Melanoma Following Failure of PD-1 Inhibitor Treatment
25 Feb 2019 CRISPR Therapeutics and Vertex Announce Progress in Clinical Development Programs for the Investigational CRISPR/Cas9 Gene-Editing Therapy CTX001

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