DNA RNA and Cells

29 Sep 2020 Intellia Therapeutics Presents New Preclinical Data Showing Persistent In Vivo Editing and Durability of Effect Following CRISPR/Cas9-Based Treatment
29 Sep 2020 Celyad Oncology Announces Clinical Trial Collaboration to Evaluate CYAD-101 with KEYTRUDA® (pembrolizumab) in Patients with Microsatellite Stable mCRC
28 Sep 2020 Sarepta Therapeutics Reports Sustained Functional Improvement Two Years After Treatment with SRP-9001, its Investigational Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy
28 Sep 2020 Sarepta Therapeutics Investigational Gene Therapy SRP-9003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2E Shows Sustained Functional Improvements 18-months After Administration
25 Sep 2020 OliX Pharmaceuticals Announces IND Submission to U.S. FDA to Evaluate the Efficacy of OLX10010 in Phase 2a Clinical Trial
25 Sep 2020 Dutch Amarna Therapeutics enters research collaboration with Spanish FPS, examining the efficacy of its SV40-based SVecTM gene delivery vector platform technology in diabetes mellitus type 1 and multiple sclerosis
25 Sep 2020 Astellas and University of Pittsburgh Announce Research Collaboration for Treatment of Dry Age-related Macular Degeneration- Striving to establish a new treatment using adeno-associated virus (AAV)-based gene therapy
25 Sep 2020 BioMarin, Pioneer in Phenylketonuria (PKU) and Gene Therapy, Doses First Participant in Global PHEARLESS Phase 1/2 Study of BMN 307 Gene Therapy
24 Sep 2020 Ardigen has received funding to accelerate its AI-driven TCR discovery platform for novel cell therapies
24 Sep 2020 Nutcracker Therapeutics Announces $60 Million Series B Financing to Advance mRNA Therapeutics Produced on Transformational Biochip-Based ACORN Platform
23 Sep 2020 U.S. Food and Drug Administration (FDA) Accepts for Priority Review Bristol Myers Squibb and bluebird bio Application for Anti-BCMA CAR T Cell Therapy Idecabtagene Vicleucel (Ide-cel, bb2121)
22 Sep 2020 City of Hope Enters Licensing Agreement With Chimeric to Develop Its Pioneering Chlorotoxin CAR T Cell Therapy
22 Sep 2020 Gyroscope Therapeutics Granted FDA Fast Track Designation for GT005, an Investigational Gene Therapy for Dry Age-Related Macular Degeneration
21 Sep 2020 T-knife and Catalent Sign Technology Transfer and Manufacturing Agreement for Autologous T-Cell Receptor-Based Cell Therapy
20 Sep 2020 Sensei Biotherapeutics Reports Early Data from Phase 1/2 Clinical Trial of SNS-301 in Combination with Pembrolizumab in Advanced Head and Neck Cancer Patients at ESMO 2020
18 Sep 2020 Precision BioSciences and Servier Expand CAR T Oncology Development Collaboration with Four New Programs Targeting Hematological and Solid Tumors
17 Sep 2020 LAVA Therapeutics Announces $83 million Series C Financing to Advance Novel Immuno-Oncology Programs
17 Sep 2020 Arrowhead ARO-AAT Phase 2 Interim Results in Patients with Alpha-1 Liver Disease Demonstrate Improvements in Key Parameters after Six Months of Treatment
16 Sep 2020 Obsidian Therapeutics Announces Bristol Myers Squibb Opt-In of cytoDRiVE™ Cell Therapy Candidate
16 Sep 2020 GenSight Biologics Submits EU Marketing Authorisation Application for LUMEVOQ® Gene Therapy to Treat Vision Loss due to Leber Hereditary Optic Neuropathy (LHON)
14 Sep 2020 Treatment with RNA-Targeting Gene Therapy Reverses Molecular and Functional Features of Myotonic Dystrophy Type 1 in Mice
14 Sep 2020 Neogene Therapeutics Raises $110 Million Series A Financing to Develop Next-Generation Fully Personalized Neo-Antigen T Cell Receptor (TCR) Therapies
12 Sep 2020 Neurocrine Biosciences and Voyager Therapeutics Present New Long-Term Three-Year Data Demonstrating that One-Time Treatment with an Investigational Gene Therapy Showed Sustained Improvement in Motor Function in Patients with Parkinson's Disease
10 Sep 2020 Immatics Provides Update on IMA204 ACTengine® Cell Therapy Program Targeting the Tumor Microenvironment
09 Sep 2020 REGENXBIO Announces Dosing of First Patient in Phase II AAVIATE™ Trial of RGX-314 for the Treatment of Wet AMD Using Suprachoroidal Delivery

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