DNA RNA and Cells

25 Jun 2022 Intellia and Regeneron Present Updated Interim Data from Phase 1 Study of CRISPR-based NTLA-2001 for the Treatment of Transthyretin (ATTR) Amyloidosis Demonstrating that Deep Serum TTR Reductions Remained Durable After a Single DoseIntellia and Regeneron
24 Jun 2022 Sarepta Therapeutics Provides Update on SRP-5051 for the Treatment of Duchenne Muscular Dystrophy
24 Jun 2022 uniQure Announces Update on Low-Dose Cohort in Phase I/II Clinical Trial of AMT-130 Gene Therapy for the Treatment of Huntington’s Disease
23 Jun 2022 Krystal Biotech Submits Biologics License Application to U.S. FDA Seeking Approval of B-VEC for the Treatment of Patients with Dystrophic Epidermolysis Bullosa
21 Jun 2022 Carbon Biosciences Launches with $38 Million Series A Financing to Advance Novel Gene Therapy Platform and Pipeline
21 Jun 2022 Kiromic BioPharma Pipeline to Prioritize a New Gamma Delta T-cell Product Candidate
21 Jun 2022 Eplontersen met co-primary and secondary endpoints in interim analysis of the NEURO-TTRansform Phase III trial for hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN)
21 Jun 2022 Acepodia Announces FDA Clearance of IND Application for ACE1831, an Anti-CD20 Armed Allogeneic gamma delta T-cell Therapy Candidate to Treat Patients with non-Hodgkin's Lymphoma
21 Jun 2022 Ionis announces eplontersen met co-primary and secondary endpoints in interim analysis of the Phase 3 NEURO-TTRansform study for hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN)
21 Jun 2022 European Medicines Agency Validates Bristol Myers Squibb’s Application for CAR T Cell Therapy Breyanzi in Relapsed or Refractory Large B-cell Lymphoma After First-Line Therapy
20 Jun 2022 Onward Therapeutics Made a Second Strategic Equity Investment in Emercell for Their NK Cell Technology in Cancer Immunotherapy
17 Jun 2022 Cellenkos Receives FDA Clearance of Investigational New Drug (IND) Application for CK0804 as Add on Therapy to Ruxolitinib for the Treatment of Myelofibrosis
14 Jun 2022 Curocell announced impressive CR rate in Phase 1 study with anbal-cel, the next generation CD19 CAR-T integrated OVIS™ platform
14 Jun 2022 Regulus Therapeutics Announces First Subject Dosed in Phase 1 Single-Ascending Dose (SAD) Clinical Trial of RGLS8429 for the Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD)
14 Jun 2022 Alnylam Announces FDA Approval of AMVUTTRA™ (vutrisiran), an RNAi Therapeutic for the Treatment of the Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis in Adults
13 Jun 2022 Gracell Biotechnologies Presents Updated Clinical Data for BCMA/CD19 Dual-targeting FasTCAR GC012F in RRMM at EHA2022 Congress, Highlighting 100% MRD Negativity Rate in All Treated Patients
13 Jun 2022 Innovent and IASO Bio Present Updated Data of BCMA CAR-T Cell Therapy (Equecabtagene Autoleucel) at EHA 2022
12 Jun 2022 CRISPR Therapeutics Presents Positive Results from its Phase 1 COBALT™-LYM Trial of CTX130™ in Relapsed or Refractory T Cell Malignancies at the 2022 European Hematology Association (EHA) Congress
11 Jun 2022 Oncternal Therapeutics Presents New Preclinical Data from its anti-ROR1 Cell Therapy Collaboration with the Karolinska Institutet at the EHA2022 Congress
11 Jun 2022 Vertex and CRISPR Therapeutics Present New Data on More Patients With Longer Follow-Up Treated With exagamglogene autotemcel (exa-cel) at the 2022 European Hematology Association (EHA) Congress
11 Jun 2022 Caribou Biosciences Reports Positive Additional Data from CB-010 Allogeneic CAR-T Cell Therapy Phase 1 ANTLER Trial at the European Hematology Association (EHA) 2022 Hybrid Congress
10 Jun 2022 Positive clinical results of ACP-001 presented at EHA2022
10 Jun 2022 Wugen Presents New Preclinical Data Demonstrating Enhanced Anti-Tumor Activity of WU-NK-101 at the European Hematology Association (EHA) 2022 Hybrid Congress
10 Jun 2022 Affimed Presents Preclinical Data of Novel Innate Cell Engager AFM28 at the Annual Meeting of the European Hematology Association (EHA)
10 Jun 2022 Seelos Therapeutics Announces Data Demonstrating Statistically Significant Downregulation of mRNA and Reduction of Alpha Synuclein in an In Vitro Gene Therapy Study of SLS-004 Utilizing CRISPR-dCas9 in Dementia with Lewy Bodies

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