DNA RNA and Cells

9 November 2023

U.S. Food and Drug Administration Accepts for Priority Review Bristol Myers Squibb’s Application for Breyanzi (lisocabtagene maraleucel)for Relapsed or Refractory Chronic Lymphocytic Leukemia (CLL) or Small Lymphocytic Lymphoma (SLL)

9 November 2023

Atara Biotherapeutics Announces Primary Analysis Data from Phase 2 EMBOLD Clinical Trial of ATA188 in Non-Active Progressive Multiple Sclerosis

8 November 2023

Grace Science Announces FDA Clearance of Investigational New Drug (IND) Application to Initiate a Phase 1/2/3 Trial for the Treatment of NGLY1 Deficiency with GS-100, an AAV9 NGLY1 Gene Therapy

8 November 2023

Nutcracker Therapeutics Demonstrates Anti-tumor Responses of Two Preclinical mRNA Drug Candidates at 2023 SITC Annual Meeting

7 November 2023

Bloomsbury Genetic Therapies Announces First Patient Enrolled in Phase 1/2 HORACE Clinical Trial of Investigational Gene Therapy BGT-OTCD for the Treatment of Ornithine Transcarbamylase Deficiency (OTCD)

7 November 2023

AviadoBio Announces FDA IND Clearance and Fast Track Designation for AVB-101 for the Treatment of Frontotemporal Dementia with Progranulin (GRN) Mutations

6 November 2023

TILT Biotherapeutics Announces Positive Clinical Data on Lead Asset TILT-123 at Society for Immunotherapy of Cancer 2023

6 November 2023

CRISPR Therapeutics Announces Preclinical Data at the American Heart Association (AHA) Scientific Sessions 2023

5 November 2023

Adverum Biotechnologies Announces 3-Year Efficacy and Safety Results from the OPTIC Extension Study in Patients with Wet AMD at AAO 2023

4 November 2023

REGENXBIO Presents Positive One Year Data from Phase II ALTITUDE® Trial of ABBV-RGX-314 for Treatment of Diabetic Retinopathy Using Suprachoroidal Delivery

4 November 2023

InGeneron Announces Encouraging Results from Emory University’s 480-Patient Phase III Knee Osteoarthritis Trial

3 November 2023

Candel Therapeutics Announces Initial Positive Interim Data from Randomized Phase 2 Clinical Trial of CAN-2409 in Non-Metastatic Pancreatic Cancer

3 November 2023

Allogene Therapeutics Presents Preclinical Data on a Novel Allogeneic CAR T Product Candidate Targeting Claudin18.2 at the Society for Immunotherapy of Cancer Annual Meeting

3 November 2023

Actym to Present Preclinical Data on Clinical Candidate ACTM-838 at 38th SITC Annual Meeting

3 November 2023

MiNK Therapeutics Presents Clinical Activity and Long-Term Persistence of Allogeneic iNKT Cells in Solid Tumors at SITC 2023

2 November 2023

Huidagene Therapeutics announces first patient dosed in multinational phase 1/2 trial of HG004 for inherited blindness

2 November 2023

Silence Therapeutics Announces Positive Topline Results from Phase 1 Multiple Dose Study of Zerlasiran in Subjects with High Lipoprotein(a) and Stable Atherosclerotic Cardiovascular Disease

2 November 2023

4DMT Presents Positive Interim Data from Phase 1/2 AEROW Clinical Trial of Aerosolized 4D-710 for Cystic Fibrosis at 2023 NACFC

1 November 2023

Cellectis Presents Pre-Clinical Data on Multi-armored Allogeneic MUC1-CAR T-cells Targeting Triple-Negative Breast Cancer at the Society for Immunotherapy of Cancer (SITC) 38th Annual Meeting

1 November 2023

Cellectis Announces Strategic Collaboration and Investment Agreements with AstraZeneca

1 November 2023

Human Biome Announces Prediction of first-in class, Biosynthetic Microbiome Against Silent Pandemic of Antibiotic-Resistant Bacteria and Doses First Patients in Contextual FMT Study to validate it

1 November 2023

Be Biopharma Presents Preclinical Data Demonstrating Ex-Vivo Engineered B Cell Medicines Produce Highly Active and Sustained Levels of Antitumor Biologics

31 October 2023

GSK enters agreement to obtain exclusive license for JNJ-3989 to expand the development of bepirovirsen

31 October 2023

Chimeric Therapeutics announces FDA clearance of IND application for CHM 2101, a novel CDH17 CAR T cell therapy for advanced gastrointestinal cancers

31 October 2023

Roche announces EMBARK trial in Duchenne muscular dystrophy (DMD) did not reach primary endpoint, but shows positive efficacy outcomes on all timed functional key endpoints

31 October 2023

Sarepta Therapeutics Announces Topline Results from EMBARK, a Global Pivotal Study of ELEVIDYS Gene Therapy for Duchenne Muscular Dystrophy

30 October 2023

Prime Medicine Presents First-ever Prime Editing Data in Non-human Primates Demonstrating Highly Efficient Ability of Prime Editors to Precisely Correct Disease-causing Mutation of GSD1b

27 October 2023

First Clinical Results of ATA-100, a Gene Therapy for the Treatment of Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9), Presented at ESGCT

27 October 2023

TransCode Therapeutics Announces Prolonged Survival in Murine Models with Glioblastoma Treated with Its Lead Candidate, TTX-MC138

27 October 2023

Regeneron Shares Preliminary Results Showing Gene Therapy Improves Auditory Responses in Child with Profound Genetic Hearing Loss

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