DNA RNA and Cells

12 Jul 2019 GenSight Biologics completes enrollment of GS010 REFLECT Phase III trial in the treatment of Leber Hereditary Optic Neuropathy ahead of schedule
11 Jul 2019 BioTime Initiates Dosing in Phase I/IIa Clinical Study of OpRegen for Treatment of Dry-AMD Utilizing Orbit Subretinal Delivery System
08 Jul 2019 Bellicum Pharmaceuticals Announces Rivo-cel™ Achieves Primary Endpoint in Pediatric Registrational Trial
08 Jul 2019 BioMarin Plans Regulatory Submissions for Marketing Authorization of Valoctocogene Roxaparvovec to Treat Severe Hemophilia A in 4Q 2019 in both U.S. and Europe
08 Jul 2019 Casebia Highlights Novel Research in Gene Editing Therapeutics for Hemophilia A
08 Jul 2019 Horizon Discovery’s SMARTvector shRNA Technology Deployed in Celyad’s Successful IND Filing for Next-Generation CAR-T Cell Therapy, CYAD-02
08 Jul 2019 Celyad Presents Update on Autologous & Allogeneic NKG2D-based CAR-T Therapies in Solid Tumors
06 Jul 2019 uniQure Announces 36 Weeks of Follow-Up Data from Phase IIb Study of AMT-061 and Long-Term Follow-Up Data for AMT-060 in Patients with Hemophilia B
06 Jul 2019 Sangamo and Pfizer Announce Updated Phase 1/2 Results for SB-525 Investigational Hemophilia A Gene Therapy Showing Sustained Increased Factor VIII Levels
04 Jul 2019 Infant Bacterial Therapeutics has recruited the first patient in the pivotal Phase III clinical study
03 Jul 2019 Unum Therapeutics Announces Regulatory Update from Phase 1 Trial with ACTR087
01 Jul 2019 Alnylam Submits Marketing Authorization Application to the European Medicines Agency for Givosiran for the Treatment of Acute Hepatic Porphyria
01 Jul 2019 Semma Therapeutics Announces Pre-Clinical Proof-of-Concept in Two Lead Programs in Type 1 Diabetes
01 Jul 2019 Vedanta Biosciences Announces First Patient Enrolled in VE416 Phase 1b/2 Food Allergy Clinical Study
01 Jul 2019 Pfizer Presents Initial Clinical Data on Phase 1b Gene Therapy Study for Duchenne Muscular Dystrophy (DMD)
28 Jun 2019 Ziopharm Oncology Initiates Phase 2 Trial Evaluating Combination Therapy of Controlled IL-12 with Libtayo® (cemiplimab-rwlc) to Treat Patients with Recurrent Glioblastoma
28 Jun 2019 Alexion Receives FDA Approval of SOLIRIS® (eculizumab) for the Treatment of Adults with Neuromyelitis Optica Spectrum Disorder (NMOSD) who are Anti-Aquaporin-4 (AQP4) Antibody Positive
27 Jun 2019 Assembly Biosciences Announces First Patient Dosed with ABI-M201 in Phase 1b Clinical Trial for Mildly to Moderately Active Ulcerative Colitis
27 Jun 2019 Wave Life Sciences Announces Initiation of DYSTANCE 51, a Phase 2/3 Clinical Trial of Suvodirsen
27 Jun 2019 Translate Bio Announces FDA Clearance to Proceed with a Single-ascending Dose (SAD) Phase 1/2 Clinical Trial for Ornithine Transcarbamylase (OTC) Deficiency
27 Jun 2019 Precigen Announces First Patient Dosed in Phase 1/1b Study of PRGN-3006 UltraCAR-T™ in Patients with Relapsed or Refractory Acute Myeloid Leukemia (AML) or Higher Risk Myelodysplastic Syndrome (MDS)
25 Jun 2019 TILT Biotherapeutics Initiates Collaboration With Merck KGaA, Darmstadt, Germany and Pfizer Investigating the Combination of Oncolytic Virus, TILT-123, And Anti-PDL1 Antibody, Avelumab
24 Jun 2019 NantKwest Launches First-in-class, First-in-human Phase I Clinical Trial With a Targeted PD-L1 t-haNK Cell Therapy in Patients With Solid Tumors
24 Jun 2019 Minerva Biotechnologies Announces FDA Acceptance of IND Application for huMNC2-CAR44 T cells to Treat Metastatic Breast Cancer
24 Jun 2019 Krystal Biotech Announces Positive Results from Phase 2 Clinical Trial (“GEM-2 study”) of KB103 and Receives Regenerative Medicine Advanced Therapy (“RMAT”) Designation from FDA for KB103

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