Reverse transcriptase

Rznomics Inc. Secures Orphan Drug Designation from FDA for RZ-001 in Hepatocellular Carcinoma

Retrieved on: 
Friday, February 2, 2024
GBM, RNA, Therapy, Tetrahymena, Patient, Safety, HCC, Mutation, FDA, Marketing, Messenger, MFDS, Reverse transcriptase, Hepatocellular carcinoma, ODD, Pharmaceutical industry

SEONGNAM, South Korea, Feb. 2, 2024 /PRNewswire/ -- Rznomics Inc., a South Korea based biopharmaceutical company specialized in the development of RNA-based gene therapeutics, has received an Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for RZ-001, for the treatment of patients with Hepatocellular carcinoma (HCC).

Key Points: 
  • SEONGNAM, South Korea, Feb. 2, 2024 /PRNewswire/ -- Rznomics Inc., a South Korea based biopharmaceutical company specialized in the development of RNA-based gene therapeutics, has received an Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for RZ-001, for the treatment of patients with Hepatocellular carcinoma (HCC).
  • This also may provide access to specialized regulatory assistance from FDA's Office of Orphan Products Development (OOPD).
  • "This FDA Orphan Drug Designation further underlines the potential of our pipeline to expeditiously address the current unmet medical needs of patients with Hepatocellular Carcinoma," said Seong-Wook Lee, CEO and founder of Rznomics.
  • Most recently, Rznomics has entered into an HCC clinical collaboration agreement with F.Hoffmann-La Roche Ltd (Roche) to study RZ-001, in combination with Roche's atezolizumab.

Rznomics Inc. Secures Orphan Drug Designation from FDA for RZ-001 in Hepatocellular Carcinoma

Retrieved on: 
Friday, February 2, 2024
GBM, RNA, Therapy, Tetrahymena, Patient, Safety, HCC, Mutation, FDA, Marketing, Messenger, MFDS, Reverse transcriptase, Hepatocellular carcinoma, ODD, Pharmaceutical industry

SEONGNAM, South Korea, Feb. 2, 2024 /PRNewswire/ -- Rznomics Inc., a South Korea based biopharmaceutical company specialized in the development of RNA-based gene therapeutics, has received an Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for RZ-001, for the treatment of patients with Hepatocellular carcinoma (HCC).

Key Points: 
  • SEONGNAM, South Korea, Feb. 2, 2024 /PRNewswire/ -- Rznomics Inc., a South Korea based biopharmaceutical company specialized in the development of RNA-based gene therapeutics, has received an Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for RZ-001, for the treatment of patients with Hepatocellular carcinoma (HCC).
  • This also may provide access to specialized regulatory assistance from FDA's Office of Orphan Products Development (OOPD).
  • "This FDA Orphan Drug Designation further underlines the potential of our pipeline to expeditiously address the current unmet medical needs of patients with Hepatocellular Carcinoma," said Seong-Wook Lee, CEO and founder of Rznomics.
  • Most recently, Rznomics has entered into an HCC clinical collaboration agreement with F.Hoffmann-La Roche Ltd (Roche) to study RZ-001, in combination with Roche's atezolizumab.

Rznomics Inc. Secures Fast Track Designation from the U.S. FDA for RZ-001

Retrieved on: 
Friday, November 10, 2023
Priority review, Korean Ministry of Culture, Safety, MFDS, Cancer, GBM, Clinical trial, Mortality, Fast track (FDA), Patient, Reverse transcriptase, Central nervous system, FDA, Survival rate, Sympathetic nervous system, Messenger, Therapy, Accelerated approval (FDA), RNA, Pharmaceutical industry, Medical device, Food, Glioblastoma

Fast Track Designation is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and address unmet medical needs, thus enabling drugs to reach patients sooner.

Key Points: 
  • Fast Track Designation is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and address unmet medical needs, thus enabling drugs to reach patients sooner.
  • If relevant criteria are met, the drug with Fast Track Designation may also be eligible for Accelerated Approval and Priority Review.
  • GBM is known as the most malignant tumor in Central Nervous system with high mortality rate but lacks effective therapies.
  • "We are proud to receive Fast Track Designation from the FDA," said Dr. Seong-Wook Lee, Chief Executive Officer of Rznomics.

Rznomics Inc. Secures Fast Track Designation from the U.S. FDA for RZ-001

Retrieved on: 
Friday, November 10, 2023
Priority review, Korean Ministry of Culture, Safety, MFDS, Cancer, GBM, Clinical trial, Mortality, Fast track (FDA), Patient, Reverse transcriptase, Central nervous system, FDA, Survival rate, Sympathetic nervous system, Messenger, Therapy, Accelerated approval (FDA), RNA, Pharmaceutical industry, Medical device, Food, Glioblastoma

Fast Track Designation is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and address unmet medical needs, thus enabling drugs to reach patients sooner.

Key Points: 
  • Fast Track Designation is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and address unmet medical needs, thus enabling drugs to reach patients sooner.
  • If relevant criteria are met, the drug with Fast Track Designation may also be eligible for Accelerated Approval and Priority Review.
  • GBM is known as the most malignant tumor in Central Nervous system with high mortality rate but lacks effective therapies.
  • "We are proud to receive Fast Track Designation from the FDA," said Dr. Seong-Wook Lee, Chief Executive Officer of Rznomics.

Lineage Cell Therapeutics and Cancer Research UK Report Topline Phase 1 Study Results With VAC2 for the Treatment of Advanced Non-small Cell Lung Cancer

Retrieved on: 
Monday, July 24, 2023
Biotechnology, Health, Pharmaceutical, Clinical Trials, Oncology, Exercise, Injection, BS, FRCP, VAC, Vaccination, Reverse transcriptase, Neoplasm, Cancer research, Survival, Geographic atrophy, Cancer, Lung cancer, Cancer Research UK, Research, Doctor of Philosophy, ID, Trial of the century, Șaeș, Publication, MD, Patient, University, TASE, University of Birmingham, NYSE American, Drug development, VAC2, Toxic epidermal necrolysis, Lineage Cell Therapeutics, Immunology, Therapy, Disease, Safety, Vaccine, Medical imaging, Lineage, Immunotherapy, NSCLC

All eight subjects enrolled and treated completed the full per protocol vaccination regimen, which consisted of six-consecutive weekly intradermal (ID) injections of 1 x 107 viable VAC2 cells.

Key Points: 
  • All eight subjects enrolled and treated completed the full per protocol vaccination regimen, which consisted of six-consecutive weekly intradermal (ID) injections of 1 x 107 viable VAC2 cells.
  • Overall, VAC2 was well-tolerated, there were no unexpected SAEs, and there were no dose limiting toxicities.
  • Brian Culley, Chief Executive Officer of Lineage, added: “The conclusion of this partnered study represents an important milestone for Lineage’s allogeneic cell therapy pipeline.
  • Lineage, Cancer Research UK, and the participating investigators intend to present these data at future medical and scientific conferences and submit publications to relevant journals for peer review.

Geron Announces New Data and Analyses from IMerge Phase 3 Presented at EHA Reporting Robust Durability of Transfusion Independence, Evidence of Disease-Modifying Activity and Favorable Fatigue PRO in Imetelstat-Treated Lower Risk MDS Patients

Retrieved on: 
Monday, June 12, 2023
Biotechnology, FDA, Other Health, Health, Pharmaceutical, Oncology, Other Science, Research, Hospitals, Science, Clinical Trials, Neutropenia, FACIT, RBC, TET2, University, MOA, Telomerase, ESA, Reverse transcriptase, TI, Erythropoiesis-stimulating agent, Chromosome, Bone marrow, Neuropsychological test, Mutation, TA, Publication, MF, VAF, MDS, ASXL1, Patient, Risk, Anemia, EHA, Therapy, Biology, University of Florence, Cochran–Mantel–Haenszel statistics, Gene, Thrombocytopenia, News, IPSS, Fatigue, Disease, Silviculture, Medical device, Pharmaceutical industry, Nursing, DNMT3A, SF3B1, Imetelstat

This is the first Phase 3 trial we know of to show an improvement in fatigue in lower risk MDS patients,” stated Dr. Platzbecker.

Key Points: 
  • This is the first Phase 3 trial we know of to show an improvement in fatigue in lower risk MDS patients,” stated Dr. Platzbecker.
  • For patients treated with imetelstat, there was a numerically higher percentage of patients reporting any episode of sustained meaningful improvement in fatigue.
  • Further, patients receiving imetelstat experienced a shorter median time to first sustained clinically meaningful improvement in fatigue vs placebo (28.3 vs 65.0 weeks).
  • In addition to these IMerge Phase 3 presentations, Geron collaborators presented a translational analysis from a subset of IMerge Phase 2 patients, as well as imetelstat myelofibrosis (MF) pre-clinical results.

RZNOMICS Inc. received FDA approval to initiate clinical development of trans-splicing ribozyme-based RNA editing technology in Glioblastoma Multiforme Patients

Retrieved on: 
Friday, May 19, 2023
Therapy, GBM, Alzheimer's disease, Reverse transcriptase, Safety, Retinitis pigmentosa, Mutation, Glioblastoma, RNA, Messenger, U.S. FDA, Patient, Tetrahymena, Retinitis, Toxic Small RNA, Mall of America, IND, HCC, Vaccine

Ribozyme-based RNA editing technology developed by Rznomics has unique features, differentiating it from other nucleic acid-based editing approaches, as follows: (1) A single RNA molecule is catalytically capable of both suppressing target RNA expression and simultaneously expressing a therapeutic RNA.

Key Points: 
  • Ribozyme-based RNA editing technology developed by Rznomics has unique features, differentiating it from other nucleic acid-based editing approaches, as follows: (1) A single RNA molecule is catalytically capable of both suppressing target RNA expression and simultaneously expressing a therapeutic RNA.
  • (2) Safety can be improved by selectively inducing therapeutic RNA expression only in cells/tissues where the target gene is expressed.
  • Through the advanced development phase, I hope Rznomics can provide more new therapeutic options to patients suffering from intractable diseases.
  • In addition to the HCC & GBM, Rznomics is developing ribozyme-based RNA editing treatments for Alzheimer's disease (RZ-003) and inherited retinal dystrophies, called Retinitis pigmentosa (RZ-004).

RZNOMICS Inc. received FDA approval to initiate clinical development of trans-splicing ribozyme-based RNA editing technology in Glioblastoma Multiforme Patients

Retrieved on: 
Friday, May 19, 2023
Therapy, GBM, Alzheimer's disease, Reverse transcriptase, Safety, Retinitis pigmentosa, Mutation, Glioblastoma, RNA, Messenger, U.S. FDA, Patient, Tetrahymena, Retinitis, Toxic Small RNA, Mall of America, IND, HCC, Vaccine

Ribozyme-based RNA editing technology developed by Rznomics has unique features, differentiating it from other nucleic acid-based editing approaches, as follows: (1) A single RNA molecule is catalytically capable of both suppressing target RNA expression and simultaneously expressing a therapeutic RNA.

Key Points: 
  • Ribozyme-based RNA editing technology developed by Rznomics has unique features, differentiating it from other nucleic acid-based editing approaches, as follows: (1) A single RNA molecule is catalytically capable of both suppressing target RNA expression and simultaneously expressing a therapeutic RNA.
  • (2) Safety can be improved by selectively inducing therapeutic RNA expression only in cells/tissues where the target gene is expressed.
  • Through the advanced development phase, I hope Rznomics can provide more new therapeutic options to patients suffering from intractable diseases.
  • In addition to the HCC & GBM, Rznomics is developing ribozyme-based RNA editing treatments for Alzheimer's disease (RZ-003) and inherited retinal dystrophies, called Retinitis pigmentosa (RZ-004).

Geron Corporation Reports Fourth Quarter and Full Year 2022 Financial Results and Upcoming Milestones

Retrieved on: 
Thursday, March 16, 2023
Health, Clinical Trials, Research, Pharmaceutical, Science, Biotechnology, Mutation, Lineage Cell Therapeutics, Imetelstat, ESA, Webcast, Fast Track, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Risk, HMA, Exercise, Survival, Depreciation, OS, MDS, PRO, Acute myeloid leukemia, MAA, Patient, Reverse transcriptase, Anemia, Corporation, Asterias, FDA, GAAP, Cytogenetics, Bone marrow, MD Anderson Cancer Center, Geron Corporation, IMPRESS, Safety, Clinical trial, Telomerase, AML, Food, Intellectual property, NDA, MF, Pharmaceutical industry, Vaccine, Risk management, Cryptocurrency, EU

Geron Corporation (Nasdaq: GERN), a late-stage clinical biopharmaceutical company developing a first-in-class telomerase inhibitor, imetelstat, to treat hematologic malignancies, today reported business updates, upcoming milestones and financial results for the fourth quarter and year ended December 31, 2022.

Key Points: 
  • Geron Corporation (Nasdaq: GERN), a late-stage clinical biopharmaceutical company developing a first-in-class telomerase inhibitor, imetelstat, to treat hematologic malignancies, today reported business updates, upcoming milestones and financial results for the fourth quarter and year ended December 31, 2022.
  • In addition, the Company received $59.8 million upon the cash exercise of outstanding warrants in January and February 2023.
  • For the fourth quarter of 2022, the Company reported a net loss of $42.6 million, or $0.10 per share, compared to $32.0 million, or $0.10 per share, for the fourth quarter of 2021.
  • ET on Thursday, March 16, 2023 to discuss business updates, expected upcoming milestones and fourth quarter and full year 2022 financial results.

Tessera Therapeutics Highlights Advancements Across its Gene Writing™ and Delivery Platforms Including Proof of Concept Data in Non-Human Primates

Retrieved on: 
Monday, January 9, 2023
Phenylketonuria, Genome, Cell, Gene, PST, Flagship Pioneering, Therapy, Tissue, Biochemistry, AAV, Biotechnology, Health, DNA, PKU, Mutation, LNP, Conference, Stem cell, Disease, Hepatectomy, Exon, CAR, NHP, Allele, RNA transfection, Liver, Reverse transcriptase, Genetic distance, Sickle cell disease, Vaccine, Tessera

“Our preclinical studies demonstrate that we can deliver our Gene Writers to the right location, in the liver and beyond, and make therapeutic alterations to the genome.

Key Points: 
  • “Our preclinical studies demonstrate that we can deliver our Gene Writers to the right location, in the liver and beyond, and make therapeutic alterations to the genome.
  • The Company’s proprietary non-viral lipid nanoparticles (LNPs) are designed to deliver Gene Writers™ to targeted tissues, potentially enabling the application of Gene Writing™ therapies in vivo.
  • DNA Gene Writers™ are based on recombinase or transposase element biochemistry and have been engineered to integrate a therapeutically relevant payload of choice.
  • Additional data demonstrate the ability to write longer sequences and entire genes with high efficiency and specificity.