DNA RNA and Cells

12 May 2021 Orchard's OTL-101 gene therapy offers potential cure for children with rare immunodeficiency
11 May 2021 M6P Therapeutics Presents M002 Gene Therapy Preclinical Proof-of-Concept Data for the Treatment of Mucolipidosis Type II at the ASGCT 24th Annual Meeting
11 May 2021 Tevogen Bio’s TVGN-489, an Allogeneic SARS-CoV-2 Specific Cytotoxic T Cell Therapy, Demonstrates Strong Anti-Viral Activity in Preclinical Study, as Presented by Professor Neal Flomenberg of Thomas Jefferson University
11 May 2021 First Preclinical Data from Adaptimmune’s Mesothelin HiT Program at ASGCT Demonstrate Antigen-specific Tumor Cell Killing in vitro and Complete Tumor Regression in an Animal Model
10 May 2021 Smart Immune Announces FDA Orphan Drug Status, IND Acceptance and Fast-Track Designation to Commence Phase 1/2 Clinical Trial of Proprietary Allogeneic T cell Progenitor Product SMART 101 (ProTcell™) for AML and ALL
10 May 2021 Mustang Bio Announces FDA Acceptance of IND Application for MB-106, a CD20-Targeted CAR T Therapy
07 May 2021 CRISPR Therapeutics and Nkarta Announce Global Collaboration to Develop Gene-Edited Cell Therapies for Cancer
05 May 2021 Checkmate Pharmaceuticals Announces Initiation of Patient Dosing in Potential Registration Trial of Vidutolimod (CMP-001) in Patients with Anti-PD-1 Refractory Advanced Melanoma
05 May 2021 Genascence Announces Completion of Dosing and Interim Safety Data for Phase 1 Clinical Trial of GNSC-001 in Osteoarthritis
05 May 2021 Stoke Therapeutics Presents New Data That Demonstrate Tango Antisense Oligonucleotides (ASOs) Increase OPA1 Protein Production and Improve Mitochondrial Function in Cells Derived From Patients With Autosomal Dominant Optic Atrophy (ADOA)
04 May 2021 Sarepta Therapeutics Reports Positive Clinical Results from Phase 2 MOMENTUM Study of SRP-5051 in Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 51
02 May 2021 Adverum Biotechnologies Presents Long-term Data through March 10, 2021 from the OPTIC Trial of ADVM-022 Intravitreal Gene Therapy in Treatment-experienced Wet AMD Patients at ARVO 2021
30 Apr 2021 PYC Therapeutics Highlights Continued Progress of Ocular and CNS Pipeline Programs and U.S. Expansion in First Quarter Update
30 Apr 2021 Legend Biotech Announces Submission of European Marketing Authorisation Application for BCMA CAR-T Therapy Ciltacabtagene Autoleucel (cilta-cel) for the Treatment of Relapsed and/or Refractory Multiple Myeloma
29 Apr 2021 Adverum Biotechnologies Provides Update on the INFINITY Trial Evaluating ADVM-022 in Patients with Diabetic Macular Edema
28 Apr 2021 Homology Medicines Announces New Approach to Leverage AAVHSC Platform by Delivering One-Time In Vivo Gene Therapy to Produce Antibodies in Humanized Murine Model
28 Apr 2021 Nkarta Receives NKX019 IND Clearance from U.S. Food and Drug Administration for Treatment of Relapsed/Refractory B Cell Malignancies
28 Apr 2021 Arrowhead Announces Improvement in Fibrosis after ARO-AAT Treatment in Patients with Alpha-1 Liver Disease
26 Apr 2021 uniQure Announces FDA Removes Clinical Hold on Hemophilia B Gene Therapy Program
26 Apr 2021 Scopus BioPharma Announces IND Submission to FDA for Lead Drug Candidate
26 Apr 2021 Voyager Therapeutics Receives FDA Clearance of IND Application for Gene Therapy Candidate VY-HTT01 for Treatment of Huntington’s Disease
21 Apr 2021 Brii Biosciences, Vir Biotechnology, and VBI Vaccines Announce Initiation of Phase 2 Clinical Trial of BRII-835 (VIR-2218) in Combination with BRII-179 (VBI-2601) for the Treatment of Hepatitis B
21 Apr 2021 Apic Bio Announces FDA Clearance of IND Application for Lead Gene Therapy Candidate APB-102 for the Treatment of SOD1 ALS
20 Apr 2021 Ionis initiates pivotal clinical study of novel antisense medicine to treat patients with Alexander disease
20 Apr 2021 Alnylam Presents Positive Results from HELIOS-A Phase 3 Study of Investigational Vutrisiran

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