DNA RNA and Cells

26 Sep 2022 Pfizer and Sangamo therapeutics announce phase 3 trial of investigational gene therapy for hemophilia a has re-opened recruitment
23 Sep 2022 Ionis provides update on development program evaluating PCSK9 antisense medicine for the treatment of hypercholesterolemia
22 Sep 2022 Neurona Therapeutics Presents Clinical Case Study from First Human Trial of Regenerative Cell Therapy, NRTX-1001, in Adults with Drug-Resistant Focal Epilepsy
21 Sep 2022 Persephone Biosciences Announces First Participant Enrolled in the ARGONAUT Study of Gut Microbiome-Linked Immune Modulation in Cancer Treatment Response
21 Sep 2022 Cellenkos Receives FDA Clearance of Investigational New Drug (IND) Application for CK0803 for the Treatment of Amyotrophic Lateral Sclerosis (ALS)
21 Sep 2022 Coeptis Therapeutics Enters Exclusive License Agreement with University of Pittsburgh for Rights to SNAP-CAR, a "Multi-Antigen" CAR T Technology
21 Sep 2022 CARsgen Presents North America Phase 2 Updates for CT053 BCMA CAR T at the 7th Annual CAR-TCR Summit
21 Sep 2022 Immunis Initiates Patient Enrollment for Muscle Atrophy Phase 1/2a Clinical Trial
21 Sep 2022 Decibel Therapeutics Announces Submission of Investigational New Drug (IND) Application for Lead Gene Therapy Candidate DB-OTO
21 Sep 2022 Wave Life Sciences Announces Positive Update from Phase 1b/2a SELECT-HD Trial with Initial Results Indicating Allele-Selective Target Engagement with WVE-003 in Huntington’s Disease
21 Sep 2022 Verismo Therapeutics Announces FDA Clearance of IND Application for SynKIR-110™, a KIR-CAR T Cell Immunotherapy Candidate
21 Sep 2022 Cellevolve Bio Submits Investigational New Drug (IND) Application for Global Phase 2, Placebo-controlled Study of CE-VST01-JC for the Treatment of Progressive Multifocal Leukoencephalopathy (PML)
21 Sep 2022 Alnylam Receives Approval in Europe for AMVUTTRA® (vutrisiran) for the Treatment of Hereditary Transthyretin-mediated (hATTR) Amyloidosis in Adult Patients with Stage 1 or Stage 2 Polyneuropathy
21 Sep 2022 Artiva Biotherapeutics Announces FDA Allowance of IND for AB-201, a HER2-Targeted CAR-NK for the Treatment of Solid Tumors
17 Sep 2022 bluebird bio Receives FDA Accelerated Approval for SKYSONA® Gene Therapy for Early, Active Cerebral Adrenoleukodystrophy (CALD)
17 Sep 2022 Intellia and Regeneron Announce Initial Data from the Cardiomyopathy Arm of Ongoing Phase 1 Study of NTLA-2001, an Investigational CRISPR Therapy for the Treatment of Transthyretin (ATTR) Amyloidosis
17 Sep 2022 Intellia Therapeutics Announces Positive Interim Clinical Data for its Second Systemically Delivered Investigational CRISPR Candidate, NTLA-2002 for the Treatment of Hereditary Angioedema (HAE)
16 Sep 2022 Alnylam and Regeneron Report Promising Data from Ongoing Phase 1 Study of ALN-HSD in NASH Patients and Healthy Volunteers
15 Sep 2022 AVROBIO Announces Neuronopathic Mucopolysaccharidosis Type II (nMPS-II) or Hunter Syndrome Clinical Trial Application (CTA) Accepted by U.K. Regulatory Agency to Initiate Pediatric Phase 1/2 Gene Therapy Trial
15 Sep 2022 Capstan Therapeutics Launches with $165 Million to Deliver on the Clinical Promise of Precise In Vivo Cell Engineering
13 Sep 2022 Akouos Receives FDA Clearance of its IND Application for AK-OTOF, a Gene Therapy Intended for the Treatment of OTOF-mediated Hearing Loss
13 Sep 2022 Regulus Therapeutics Announces Positive Topline Safety and Pharmacokinetic (PK) Data from the Phase 1 Single-Ascending Dose (SAD) Clinical Trial of RGLS8429 for the treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD)
12 Sep 2022 Triumvira Immunologics Presents Initial HER2-Positive Solid Tumor Clinical Data at ESMO
12 Sep 2022 Transgene Confirms the Potential of the Intravenous Route of its Invir.IO™ Oncolytic Viruses against Solid Tumors with TG6002 Phase I Data Presented at ESMO Congress 2022
11 Sep 2022 Adaptimmune Reports Positive Data in its SURPASS Trial; Outlines Plans for Advanced Clinical Development in Multiple MAGE-A4 Positive Solid Tumors

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