DNA RNA and Cells

16 Sep 2019 Alnylam Pharmaceuticals Announces Initiation of APOLLO-B Phase 3 Study of Patisiran for the Treatment of Transthyretin Amyloidosis with Cardiomyopathy
16 Sep 2019 Arrowhead Pharmaceuticals Presents Initial Top-Line Clinical Data and Preclinical Data on RNAi Candidates ARO-APOC3 and ARO-ANG3
16 Sep 2019 Positive Phase 1 Results of AKCEA-TTR-LRx Presented at the Heart Failure Society of America Annual Meeting
14 Sep 2019 Atara Biotherapeutics Reports Early Findings of Potential Efficacy from Phase 1 Study of ATA188 in Patients with Progressive Multiple Sclerosis at ECTRIMS 2019
12 Sep 2019 Adverum Biotechnologies Reports Positive 24-Week Data from First Cohort of OPTIC Phase 1 Trial of ADVM-022 Intravitreal Gene Therapy to Treat Wet AMD
12 Sep 2019 Gracell Announces Interim Readout of Investigation for First-in-class FasT CAR-19 in patients with Relapsed or Refractory B-cell Acute Lymphoblastic Leukemia and Extension in Use of FasT CAR to Other Immune Cell Programs
12 Sep 2019 Momotaro-Gene Announces First Patient Dosed in Phase 2 Clinical Trial of MTG201 in Combination with Nivolumab in Patients with Relapsed Malignant Pleural Mesothelioma
12 Sep 2019 Moderna Announces Positive Phase 1 Results for the First Systemic Messenger RNA Therapeutic Encoding a Secreted Protein (mRNA-1944)
10 Sep 2019 Passage Bio Announces New Gene Therapy Development Program for Patients with Charcot-Marie-Tooth Neuropathy Type 2A
10 Sep 2019 Translate Bio Announces Pipeline Program Update
05 Sep 2019 Rocket Pharmaceuticals Announces First Patient Treated in Phase 1/2 Registrational Trial of RP-L201 for LAD-I
05 Sep 2019 Krystal Biotech Announces Initiation of Phase 1/2 Study of KB105 in Transglutaminase-1 (“TGM1”) Deficient Autosomal Recessive Congenital Ichthyosis (“ARCI”)
05 Sep 2019 Nkarta Therapeutics Raises $114 Million in Series B Financing to Advance Multiple Programs into Clinical Trials
04 Sep 2019 Ultragenyx Announces Positive Data from Phase 1/2 Study of DTX401 Gene Therapy in Glycogen Storage Disease Type Ia
02 Sep 2019 The Medicines Company Presents Results from ORION-11, First Phase 3 Trial of Inclisiran, Showing Durable and Potent Lowering of LDL-C with Twice-Yearly Dosing
29 Aug 2019 Immunicum AB (publ) Announces Positive Phase II MERECA Topline Results Including Complete Tumor Responses in Metastatic Renal Cell Carcinoma Patients
29 Aug 2019 Arrowhead Collaborator Janssen Begins REEF-1 Phase 2b Combination Study in Patients with Chronic Hepatitis B Infection
27 Aug 2019 The Medicines Company Announces Positive Topline Results from First Pivotal Phase 3 Trial of Inclisiran
21 Aug 2019 NKMax America Initiates Phase I Clinical Trial of SNK in Patients with Psoriasis in Mexico
20 Aug 2019 Synlogic Discontinues Development of SYNB1020 to Treat Hyperammonemia
20 Aug 2019 Sarepta Therapeutics Receives Complete Response Letter from the US Food and Drug Administration for Golodirsen New Drug Application
16 Aug 2019 City of Hope Now Enrolling Patients for First-in-Human T Cell Trial for HPV-Associated Cancers
14 Aug 2019 NKMax America Doses First Subject in Phase I Trial of SNK in Patients with Refractory Cancer
14 Aug 2019 Mallinckrodt Announces Publication of Results of Phase 1b Clinical Trial of StrataGraft® Regenerative Tissue in Burns
14 Aug 2019 Renovacor Closes $11 Million Series A Financing to Advance First Gene Therapy for a Rare Cardiovascular Disease

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