DNA RNA and Cells

20 Dec 2019 Solid Biosciences Provides Data Update from SGT-001 Development Program
20 Dec 2019 Akcea Retains Rights to AKCEA-APOCIII-LRx
20 Dec 2019 NexImmune Receives IND Clearance for Phase 1/2 Trial in Relapsed / Refractory Multiple Myeloma
20 Dec 2019 Axovant Provides Clinical Program Update for AXO-AAV-GM1, a Novel Investigational Gene Therapy for GM1 Gangliosidosis
20 Dec 2019 Clinical trial application for pivotal JTA-004 Phase III study submitted
20 Dec 2019 REGENXBIO Announces Interim Data from Phase I/II Trial of RGX-121 for the Treatment of Mucopolysaccharidosis Type II (MPS II)
19 Dec 2019 Bristol-Myers Squibb Announces Submission of Biologics License Application for CAR T-Cell Therapy Lisocabtagene Maraleucel (liso-cel) to FDA
18 Dec 2019 Homology Medicines Announces Encouraging Initial Clinical Data from its pheNIX Gene Therapy Trial for PKU
17 Dec 2019 Alnylam Reports Positive Topline Results from ILLUMINATE-A Phase 3 Study of Lumasiran for the Treatment of Primary Hyperoxaluria Type 1
16 Dec 2019 Regulus Therapeutics Announces FDA Removal of Partial Clinical Hold for Multiple Ascending Dose Study of RGLS4326
16 Dec 2019 NantKwest and ImmunityBio Present Results of Landmark Trial of First-in-Human Natural Killer Cell Combination Immunotherapy With Durable, Complete Response Data and 78% Disease Control in Refractory Triple Negative Breast Cancer at SABCS
16 Dec 2019 Wave Life Sciences Announces Discontinuation of Suvodirsen Development for Duchenne Muscular Dystrophy
16 Dec 2019 Arrowhead Pharmaceuticals Files for Regulatory Clearance to Begin Phase 1 Study of ARO-HSD for Treatment of Alcohol and Nonalcohol Related Liver Disease
16 Dec 2019 SanBio and Sumitomo Dainippon Pharma Announce a Termination of a Joint Development and License Agreement for Regenerative Cell Medicine SB623 for Chronic Stroke in North America
13 Dec 2019 OncoSec Presents Interim Data of 28.5% Objective Response Rate (ORR) from Ongoing KEYNOTE-890 Study Evaluating TAVO™ in Combination with KEYTRUDA® for Heavily Pretreated, Late-Stage, Metastatic Triple Negative Breast Cancer (mTNBC) at the 2019 San Antonio
13 Dec 2019 Sarepta Therapeutics Announces FDA Approval of VYONDYS 53™ (golodirsen) Injection for the Treatment of Duchenne Muscular Dystrophy (DMD) in Patients Amenable to Skipping Exon 53
12 Dec 2019 Kite Submits Biologics License Application to U.S. Food and Drug Administration for Company’s Second CAR T Cell Therapy
11 Dec 2019 ProQR Announces First Patient Dosed in Phase 1/2 Aurora Trial of QR-1123 for Autosomal Dominant Retinitis Pigmentosa
11 Dec 2019 Exicure Reports Activity of AST-008 in Patients with Merkel Cell Carcinoma and Will Enroll Patients in Phase 2 Study
11 Dec 2019 Kite and Kiniksa Pharmaceuticals Announce Clinical Collaboration Evaluating Investigational Combination of Yescarta® and Mavrilimumab in Relapsed or Refractory Large B-Cell Lymphoma
11 Dec 2019 Arrowhead Pharmaceuticals Files IND for Phase 1b Study of ARO-HIF2 for Treatment of Clear Cell Renal Cell Carcinoma
11 Dec 2019 GenSight Biologics reports findings from REALITY study and REVERSE-RESCUE analysis highlighting positive benefits from LUMEVOQ™ (GS010) compared to LHON natural history
10 Dec 2019 Precision BioSciences Presents Updated Interim Clinical Data at the ASH Annual Meeting from Relapsed / Refractory NHL and B-ALL Patients Treated at Dose Levels 1 & 2 in the Ongoing Phase 1/2a Clinical Trial of PBCAR0191, a Novel CD19 Targeted Allogeneic C
10 Dec 2019 Abeona Therapeutics Cleared to Initiate Pivotal Phase 3 Clinical Trial Evaluating EB-101 Gene Therapy for Recessive Dystrophic Epidermolysis Bullosa
10 Dec 2019 Editas Medicine Announces In Vivo Proof-of-Concept Data for EDIT-301, in Development for the Treatment of Sickle Cell Disease and Beta-Thalassemia

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