DNA RNA and Cells

13 Jun 2019 Bone Therapeutics’ allogeneic cell therapy product, ALLOB, meets primary endpoints in Phase IIa study in patients undergoing a lumbar spinal fusion procedure
12 Jun 2019 Immunicum AB (publ) Announces Positive Topline Results from the Phase I/II Clinical Trial in GIST
11 Jun 2019 Ziopharm Oncology Announces FDA Clearance of IND for Sleeping Beauty TCR-T Cell Therapy Trial at NCI
10 Jun 2019 Homology Medicines Initiates Phase 1/2 Study for HMI-102 Gene Therapy Candidate for Adults with PKU and Expects Initial Clinical Results by Year End 2019
07 Jun 2019 SillaJen Announces First Patient Enrolled in Part 2 of Renal Cell Carcinoma Trial with Pexa-Vec in Combination with Regeneron's Libtayo® (cemiplimab-rwlc)
06 Jun 2019 Prevail Therapeutics Announces IND Active for Phase 1/2 Trial of its Gene Therapy PR001 to Treat Parkinson’s Disease Patients with GBA1 Mutations
06 Jun 2019 Enzyvant Announces FDA Acceptance of Biologics License Application (BLA) and Priority Review Status for RVT-802, a Novel Investigational Tissue-Based Regenerative Therapy for Pediatric Congenital Athymia
06 Jun 2019 Alnylam Completes Rolling Submission of New Drug Application to U.S. Food and Drug Administration (FDA) for Givosiran for the Treatment of Acute Hepatic Porphyria
04 Jun 2019 Allogene Therapeutics Announces FDA Clearance of the IND for ALLO-715, a BCMA Allogeneic CAR T (AlloCAR T™) Therapy
04 Jun 2019 Cellenkos, Inc. Initiates Phase 1 Clinical Trial of CK0801 for Treatment of Bone Marrow Failure Syndromes
04 Jun 2019 NantKwest Launches First-in-Human Targeted NK Phase I Clinical Trial of CD19 T-haNK Cell Therapy in Lymphoma Patients
04 Jun 2019 NantKwest Announces Durable Complete Responses in Multiple Advanced-Stage Solid Tumor Cancers Following Off-the-Shelf NK Cell Therapy in First 35 Patients
04 Jun 2019 Strand Therapeutics Announces $6 Million Seed Financing to Advance its Programmable mRNA Therapeutics Platform
03 Jun 2019 bluebird bio Announces EU Conditional Marketing Authorization for ZYNTEGLO™ (autologous CD34+ cells encoding βA-T87Q-globin gene) Gene Therapy for Patients 12 Years and Older with Transfusion-Dependent β-Thalassemia Who Do Not Have β0/β0 Genotype
02 Jun 2019 Talaris Therapeutics Presents Data Demonstrating Improved Quality of Life for Patients Treated with FCR001
02 Jun 2019 Kite Announces End of Phase 1 ZUMA-3 Results for KTE-X19 in Adult Patients with Relapsed or Refractory Acute Lymphoblastic Leukemia
01 Jun 2019 Bellicum Pharmaceuticals Provides Interim Data for BPX-601 in Patients with Metastatic Pancreatic Cancer for Presentation at ASCO Annual Meeting
01 Jun 2019 Dynavax Presents Phase 2 Data on SD-101 in Combination with KEYTRUDA® (pembrolizumab) for Patients with Head and Neck Squamous Cell Carcinoma at the 2019 ASCO Annual Meeting
31 May 2019 Alnylam Doses First Patient in Phase 1 Study of ALN-AGT, an Investigational RNAi Therapeutic for the Treatment of Hypertension
31 May 2019 Kite and Humanigen Announce Clinical Collaboration to Evaluate Investigational Combination of Yescarta® (Axicabtagene Ciloleucel) with Lenzilumab in Relapsed/Refractory Diffuse Large B-Cell Lymphoma
30 May 2019 Mesoblast initiates rolling submission of biologics license application (BLA) to U.S. FDA for remestemcel-l in the treatment of acute graft versus host disease
29 May 2019 Freeline presents preclinical data and phase 1/2 study design of the FLT190 AAV gene therapy for Fabry Disease
29 May 2019 BioMarin Announces that Phase 3 Cohort of Valoctocogene Roxaparvovec, Gene Therapy Study in Severe Hemophilia A Met Pre-Specified Criteria for Regulatory Submissions in the U.S. and Europe
28 May 2019 The Medicines Company Presents Clinical Data Analyses for Inclisiran at 87th European Atherosclerosis Society Congress
24 May 2019 AveXis receives FDA approval for Zolgensma®, the first and only gene therapy for pediatric patients with spinal muscular atrophy (SMA)

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