DNA RNA and Cells

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
13 Aug 2019 ProQR Announces Clearance of IND to Start Clinical Trial of QR-1123 in Patients with Autosomal Dominant Retinitis Pigmentosa (adRP)
12 Aug 2019 TScan Therapeutics Announces Pioneering Publication in Scientific Journal Cell Describing New Methods to Identify Targets for T Cell Receptor Immunotherapy
08 Aug 2019 Arrowhead Pharmaceuticals Doses First Patient in SEQUOIA Phase 2/3 Study of ARO-AAT for Treatment of Alpha-1 Liver Disease
07 Aug 2019 Moderna Provides Business Updates and Reports Second Quarter 2019 Financial Results
07 Aug 2019 Akcea and Ionis Report Top-line Results from the BROADEN Study with WAYLIVRA® (volanesorsen) in Patients with Familial Partial Lipodystrophy
07 Aug 2019 Novartis stands behind Zolgensma® (onasemnogene abeparvovec-xioi) for the treatment of children less than 2 years of age with spinal muscular atrophy
06 Aug 2019 Evelo Biosciences Reports Positive EDP1815 Interim Clinical Data in Psoriasis Patients at Low Dose in Ongoing Phase 1b Trial
06 Aug 2019 MOLOGEN AG Announces Top Line Data of Pivotal IMPALA Study in Metastatic Colorectal Cancer

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