DNA RNA and Cells

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
11 Sep 2022 BioNTech Presents Encouraging Phase 1/2 Follow-up Data for CAR-T Candidate BNT211 in Hard-To-Treat Solid Tumors at ESMO
11 Sep 2022 Wugen Presents Preclinical Data on WU-NK-101 in Solid Tumor Models at the European Society for Medical Oncology (ESMO) Congress 2022
09 Sep 2022 ESMO 2022: Interim Results for Treatment of Platinum-Resistant Ovarian Cancer with p62 Plasmid DNA Show Statistically Significant Changes in Progression-Free Survival
09 Sep 2022 Alnylam Presents Positive Results from the APOLLO-B Phase 3 Study of Patisiran in Patients with ATTR Amyloidosis with Cardiomyopathy
07 Sep 2022 Kite’s CAR T-cell Therapy Tecartus® Granted European Marketing Authorization for the Treatment of Relapsed or Refractory Acute Lymphoblastic Leukemia (r/r ALL)
06 Sep 2022 Dyne Therapeutics Announces First Patient Dosed in Phase 1/2 DELIVER Clinical Trial of DYNE-251 for the Treatment of Duchenne Muscular Dystrophy
06 Sep 2022 Dyne Therapeutics Announces Initiation of Phase 1/2 ACHIEVE Clinical Trial of DYNE-101 for the Treatment of Myotonic Dystrophy Type 1
06 Sep 2022 Arsenal Biosciences Closes $220 Million Series B Financing to Advance Programmable Cell Therapy Programs into Clinical Development
06 Sep 2022 Sarepta Therapeutics Announces That FDA has Lifted its Clinical Hold on SRP-5051 for the Treatment of Duchenne Muscular Dystrophy
06 Sep 2022 CellOrigin Biotech announced strategic global collaboration with Qilu Pharmaceutical to develop "off-the-shelf" CAR-iMAC cell therapy
01 Sep 2022 Immusoft Announces FDA Clearance of IND Application for ISP-001 for MPS I, the First Engineered B Cell Therapy to Enter into Clinical Trials

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