DNA RNA and Cells

25 Oct 2021 Egle Therapeutics Raises 40 Million € Series a to Develop First-In-Class T-regulatory Cells Therapies Based On Treg-starvers
25 Oct 2021 Arrowhead Files for Regulatory Clearance to Begin Phase 1/2a Study of ARO-C3 for Treatment of Complement Mediated Diseases
23 Oct 2021 Generation Bio Presents Data Demonstrating First Lipid Nanoparticle to Achieve Uniform Retinal Transduction and Tolerability via Sub-Retinal Delivery of ceDNA and mRNA at the European Society of Gene and Cell Therapy 2021 Annual Virtual Congress
23 Oct 2021 Helixmith Announces Phase 3 Study Results of Novel Gene Therapy Treatment for Diabetic Foot Ulcers at 2021 Annual Meeting of Diabetic Foot Ulcer Conference (DFCon)
21 Oct 2021 Sio Gene Therapies Announces Positive Interim Safety and Biomarker Data from Ongoing Phase 1/2 Clinical Study of AXO-AAV-GM1 Gene Therapy in GM1 Gangliosidosis
21 Oct 2021 Homology Medicines Announces Presentations Across Gene Therapy and Gene Editing Programs, including GTx-mAb, at European Society of Gene & Cell Therapy Meeting
20 Oct 2021 Voyager Therapeutics Presents Positive Preclinical Data Supporting CNS-Directed GBA1 Gene Therapy at Virtual Congress of the European Society of Gene and Cell Therapy
20 Oct 2021 Intellia Therapeutics Presents Preclinical Data Demonstrating Advancements in its Broad Genome Editing Capabilities at the 2021 European Society of Gene & Cell Therapy Annual Congress
20 Oct 2021 Krystal Biotech Announces Virtual Presentation of Pre-clinical Data on KB407 for the Treatment of Cystic Fibrosis
20 Oct 2021 Cellectis Presents Initial Preclinical Data on Two Novel Gene Therapies for Patients with RAG1 Severe Combined Immunodeficiency (SCID) and Hyper IgE syndrome at ESGCT 2021
20 Oct 2021 Dicerna Announces Results for PHYOX™4, Single-Dose Study of Nedosiran in Primary Hyperoxaluria Type 3 (PH3)
20 Oct 2021 Rocket Pharmaceuticals Announces Positive Clinical Data from RP-L201 Trial for the Treatment of LAD-I at the 28th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT)
20 Oct 2021 Chugai Announces the Decision of the Termination of Exclusive License Agreement on OBP-301 (Telomelysin), Oncolytic Viral Immunotherapy
19 Oct 2021 Boehringer Ingelheim and Partners to Accelerate Development of First-In-Class Gene Therapy for Patients with Cystic Fibrosis
18 Oct 2021 BiomX Reports Topline Results of Phase 2 Cosmetic Acne Study
18 Oct 2021 Atsena Therapeutics Unveils XLRS Gene Therapy Program Leveraging Novel Spreading Capsids
18 Oct 2021 Ultragenyx Initiates Cyprus2+, a Pivotal Clinical Trial Evaluating UX701 Gene Therapy for the Treatment of Wilson Disease
18 Oct 2021 Homology Medicines Initiates Clinical Trial for HMI-203, a One-Time Investigational Gene Therapy Candidate for Adults with MPS II (Hunter Syndrome)
18 Oct 2021 LogicBio Therapeutics Announces Early Clinical Trial Results Demonstrating First-Ever In Vivo Genome Editing in Children
18 Oct 2021 Biogen Announces Topline Results from the Tofersen Phase 3 Study and its Open-Label Extension in SOD1-ALS
18 Oct 2021 Vertex Announces Positive Day 90 Data for the First Patient in the Phase 1/2 Clinical Trial Dosed With VX-880, a Novel Investigational Stem Cell-Derived Therapy for the Treatment of Type 1 Diabetes
15 Oct 2021 Abalos Therapeutics Completes Series A Extension Bringing Total Raised to EUR 43 Million
13 Oct 2021 Regulus Therapeutics Announces Strategic Prioritization of RGLS8429, its Next-Generation Candidate for the Treatment of Autosomal Dominant Polycystic Kidney Disease
13 Oct 2021 Homology Medicines Announces World’s First Gene Editing Clinical Trial for PKU
13 Oct 2021 CRISPR Therapeutics Reports Positive Results from its Phase 1 CARBON Trial of CTX110™ in Relapsed or Refractory CD19+ B-cell malignancies

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