DNA RNA and Cells

04 Oct 2019 Sarepta Therapeutics Announces Positive Functional Results from the SRP-9003 (MYO-101) Gene Therapy Trial to Treat Limb-Girdle Muscular Dystrophy Type 2E, or Beta-Sarcoglycanopathy
04 Oct 2019 GT Biopharma Announces HIV TriKE™ Data Demonstrating NK Cell Killing of Patient HIV Infected Cells
02 Oct 2019 Dynacure Presents New Preclinical Results on Muscle Targeting with Antisense Oligonucleotides at the 24th Annual International Congress of the World Muscle Society
02 Oct 2019 NMPA Approves IND Application for CT103A, a Fully-human BCMA CAR-T for the Treatment of Relapsed/Refractory Multiple Myeloma Co-developed by IASO BIO and Innovent Biologics
02 Oct 2019 Cabaletta Bio Receives IND Clearance from FDA to Initiate First Clinical Trial of DSG3-CAART in Patients with Mucosal Pemphigus Vulgaris
02 Oct 2019 Ziopharm Oncology Announces FDA Clearance of IND for Rapid Personalized Manufacture of CD19-specific CAR-T
01 Oct 2019 Checkmate Pharmaceuticals Announces Dosing of First Patient in a Phase 1b/2 Study in Squamous Cell Carcinoma of the Head and Neck with Lead Investigational Candidate CMP-001
01 Oct 2019 Idera Pharmaceuticals Announces Initiation of the ILLUMINATE-206 Trial Evaluating Tilsotolimod in Combination with Nivolumab and Ipilimumab for the Treatment of Solid Tumors
01 Oct 2019 Clear Benefit for Patients with Synovial Sarcoma Demonstrated in Updated Data from Ongoing Phase 1 Trial with ADP-A2M4 Presented at ESMO
30 Sep 2019 Moderna Announces Open IND for Propionic Acidemia Program (mRNA-3927)
30 Sep 2019 Ocugen and CanSinoBIO Enter Strategic Partnership for Gene Therapy Co-Development and Manufacturing
27 Sep 2019 BioMarin, Pioneer in Phenylketonuria (PKU) Therapies, Submits Clinical Trial Application (CTA) in U.K. for Investigational Gene Therapy for PKU
27 Sep 2019 The Medicines Company Announces Positive Topline Results for ORION-10 Phase 3 Study of Inclisiran in ASCVD Patients
27 Sep 2019 First Chimeric Antigen Receptor (CAR) T Cell Therapy Targeting B Cell-Activating Factor Receptor (BAFF-R) Eradicates Blood Cancers That Relapsed After CD19 CAR T Therapies
25 Sep 2019 Abeona Therapeutics Provides Regulatory Update Ahead of Pivotal Phase 3 Clinical Trial for EB-101 in Recessive Dystrophic Epidermolysis Bullosa
25 Sep 2019 Kadimastem Announces Promising Interim Results of Cohort A of Its Phase 1/2a Clinical Trial in ALS
25 Sep 2019 Amphivena Closes $62 Million Series C Financing to Expand the Clinical Development of AMV564 into Solid Tumors and to Develop its T Cell Engagement Portfolio
24 Sep 2019 Topline Results of DPN Phase III Study
24 Sep 2019 Mallinckrodt Announces Positive Top-line Results from Pivotal Phase 3 Clinical Trial of StrataGraft® Regenerative Tissue in Patients with Deep Partial-thickness Thermal Burns
24 Sep 2019 GenSight Biologics Reports Sustained Efficacy and Safety at 96 Weeks in RESCUE Phase III Clinical Trial of GS010 for the Treatment of Leber Hereditary Optic Neuropathy (LHON)
22 Sep 2019 Avalon GloboCare Announces Initiation of First-in-Human Clinical Trial of its CAR-T Candidate, AVA-001, for Treatment of B-Cell Malignancies
18 Sep 2019 Procarta receives CARB-X award of up to $9.2 million
18 Sep 2019 Biogen Advances Spinal Muscular Atrophy (SMA) Clinical Research with New Study Evaluating a Higher Dose of SPINRAZA® (nusinersen) and Additional Data in a Broad Range of Patients
18 Sep 2019 Inovio Receives NIH Funding to Target its dMAb® Technology Against Antimicrobial-Resistant Infection
16 Sep 2019 Precision BioSciences Announces FDA Clearance of the IND for PBCAR20A, a CD20 Targeting Genome Edited Allogeneic CAR T Therapy

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