HAO1

Arbor Biotechnologies Presents Data Supporting Clinical Development of ABO-101 and Robust Potential of Platform to Enable Therapeutic Programs at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting

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Jeudi, mai 9, 2024
Mouse, CRISPR, Impact, Compact, Gene editing, Cell, Artificial intelligence, CGT, PCSK9, IND, CNS, TDP43, PH1, Therapy, Patient, Liver, Gene, Society, ALS, Brain cell, Central nervous system, Messenger, HAO1, NHP, Annual general meeting, Pharmacology, Engineering, Medical device

“This approach has fueled our robust pipeline of genetic medicines which address a range of liver and CNS indications, including PH1 and ALS.

Key Points: 
  • “This approach has fueled our robust pipeline of genetic medicines which address a range of liver and CNS indications, including PH1 and ALS.
  • The data show highly specific targeting of the HAO1 gene in the liver and preservation of genomic integrity upon editing.
  • Together, this strong preclinical data package and the ongoing IND-enabling studies support continued advancement of ABO-101 toward clinical evaluation.
  • Details for the presentations are as follows:
    Oral Presentation Title: Identification and Engineering of ABR-004, a Compact, High-fidelity Nuclease for Therapeutic Gene Editing

Arbor Biotechnologies to Present Data Supporting Therapeutic Programs in PH1 and ALS, and the Discovery of a Novel Type V Nuclease at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting

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Lundi, avril 22, 2024
CRISPR, Impact, Compact, Cell, CGT, PCSK9, PH1, STMN2, Liver, Gene, Society, ALS, Messenger, HAO1, NHP, Engineering, Medical device

CAMBRIDGE, Mass., April 22, 2024 (GLOBE NEWSWIRE) -- Arbor Biotechnologies®, a biotechnology company discovering and developing the next generation of genetic medicines, today announced four upcoming presentations at the 2024 American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting, taking place May 7-11 in Baltimore, Maryland.

Key Points: 
  • Arbor will present in vivo NHP data supporting clinical development of ABO-101, its most advanced gene editing therapeutic candidate designed to address primary hyperoxaluria type 1 (PH1) through inactivation of the HAO1 gene in the liver.
  • The company also will present data supporting its end-to-end nuclease development platform in a third presentation outlining the discovery and optimization of a unique, compact nuclease, termed ABR-004.
  • Proof-of-concept data in non-human primates show potent, therapeutically relevant silencing of PCSK9 in vivo with the novel nuclease, signaling opportunities for broader therapeutic applications.
  • Together, the suite of presentations will demonstrate the utility of Arbor’s proprietary discovery and development approach for enabling efficient identification and optimization of novel nucleases.

Arbor Biotechnologies Announces Presentations at the 2023 Annual Meeting of the American Society of Gene & Cell Therapy

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Mardi, mai 2, 2023
Kidney disease, Abstract, HAO1, Gene, Waste, Significant figures, American Society of Gene and Cell Therapy, Model, PH, Primary hyperoxaluria, Engineering, Annual general meeting, Cell, Oxalate, Society, Vaccine

CAMBRIDGE, Mass., May 02, 2023 (GLOBE NEWSWIRE) --  Arbor Biotechnologies®, a biotechnology company discovering and developing the next generation of genetic medicines, today announced that three abstracts were selected for poster presentation at the upcoming American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting being held from May 16-20, 2023, in Los Angeles, CA.

Key Points: 
  • CAMBRIDGE, Mass., May 02, 2023 (GLOBE NEWSWIRE) --  Arbor Biotechnologies®, a biotechnology company discovering and developing the next generation of genetic medicines, today announced that three abstracts were selected for poster presentation at the upcoming American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting being held from May 16-20, 2023, in Los Angeles, CA.
  • “Among our poster presentations, we will be providing early results with our lead product candidate, ABR-001—a novel type V CRISPR Cas, which we are developing for the treatment of primary hyperoxaluria, or PH.
  • PH is a rare, life-threatening condition that prevents the kidneys from filtering fluids and waste products from the body effectively, often resulting in end-stage renal disease.
  • We are highly encouraged by these preclinical results, which demonstrate that in vivo editing of the HAO1 gene with ABR-001 significantly reduced toxic oxalates in a mouse model of PH Type 1.

Precision BioSciences Announces Preclinical Data Showcasing Premier In Vivo Gene Editing Capabilities at American Society of Gene & Cell Therapy Annual Meeting

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Lundi, mai 16, 2022
Science, Biotechnology, Research, Pharmaceutical, Health, Infectious Diseases, Genetics, Clinical Trials, RNA transfection, HBV, Lactic acidosis, Washington Convention Center, Liver, Mitochondrial encephalomyopathy, CccDNA, Private Securities Litigation Reform Act, Animal, Mitochondrion, CAR, SEC, LNP, HAO1, Annual report, Serum, Investor, COVID-19, WT, Research, Mouse, MELAS, Growth, Achievement, DNA, Genome, Protein engineering, PH1, Infection, Insurance, PCSK9, Abstract (summary), Gene editing, Hepatitis B, Adult, Ownership, Organic acid, CTA, List, Walter E. Washington Convention Center, Hepatitis B virus, Therapy, Cell, NHP, A-DNA, PHH, Nasdaq, Ornithine transcarbamylase deficiency, Patient, Poster, OTC, Society, Privacy, HAO, Gene, AAV, Intellectual property, Forward-looking statement, Compliance, Walter, Re Recher's Will Trusts, Safety, Technology, SEC filing, Infant, Hepatocyte, Hepatitis, Mutation, Vaccine, D.C, HBsAg, IND

Data from this preclinical study demonstrate Precisions gene editing approach designed to eliminate hepatitis B virus (HBV).

Key Points: 
  • Data from this preclinical study demonstrate Precisions gene editing approach designed to eliminate hepatitis B virus (HBV).
  • These data suggest that LNP-delivered ARCUS mRNA is a promising approach and potential functional cure for chronic hepatitis B.
  • Precision will continue developing its PBGENE-HBV product candidate using LNP delivery and expects to submit an IND/CTA in 2024.
  • Preclinical data presented in this poster demonstrate Precisions gene editing approach to shift mitochondrial DNA (mtDNA) heteroplasmy for the m.3243A>G mtDNA mutation.

Alnylam Announces U.S. Food and Drug Administration Acceptance of Supplemental New Drug Application for OXLUMO® for the Treatment of Advanced Primary Hyperoxaluria Type 1

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Mardi, mars 1, 2022
FDA, Health, Genetics, Clinical Trials, Pharmaceutical, Biotechnology, Risk, Therapy, Type, ESC, Therapeutic index, European Medicines Agency, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Pain, Kidney, European Commission, Injection site reaction, Medical Affairs Bureau, Medication, Royal commission, Safety, Itch, Child, Alnylam Pharmaceuticals, Review, Erythema, Swelling, New Drug Application, Food, Adult, PH1, PDUFA, Shanda, European Directive on Traditional Herbal Medicinal Products, FDA, RNA interference, Nasdaq, EMA, Committee for Medicinal Products for Human Use, EVP, Patient, Enhance, GO, Prescription Drug User Fee Act, Milk, Committee, CHMP, Plasma, EGFR, Pregnancy, Profile, Pharmaceutical industry, Medical device, Medicine, Birth control, HAO1, Lumasiran, ILLUMINATE-C Phase 3 Study, Primary Hyperoxaluria Type 1 (PH1), RNAi, Alnylam Pharmaceuticals, LUMASIRAN, ILLUMINATE-C PHASE 3 STUDY, PRIMARY HYPEROXALURIA TYPE 1 (PH1), RNAI, ALNYLAM PHARMACEUTICALS

This filing acceptance is a positive step for patients with advanced PH1, who are at risk for the devastating complications of systemic oxalosis.

Key Points: 
  • This filing acceptance is a positive step for patients with advanced PH1, who are at risk for the devastating complications of systemic oxalosis.
  • OXLUMO is indicated for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients.
  • The most common adverse reaction that occurred in patients treated with OXLUMO was injection site reaction (38%).
  • Lumasiran is a subcutaneously administered RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) in development for the treatment of advanced primary hyperoxaluria type 1 (PH1).

Alnylam Submits Regulatory Applications to the U.S. Food and Drug Administration and European Medicines Agency to Support Label Expansion for OXLUMO® for the Treatment of Advanced Primary Hyperoxaluria Type 1

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Mardi, décembre 14, 2021
Science, Biotechnology, Research, Pharmaceutical, Health, FDA, Genetics, Clinical Trials, PH1, Dialysis, Itch, Food, Shanda, FDA, Injection site reaction, EGFR, CKD, European Medicines Agency, Pain, Child, Swelling, Alnylam Pharmaceuticals, Pregnancy, New Drug Application, Therapeutic index, ASN, Adult, Milk, Nasdaq, GO, EMA, Enhance, Hydroxyacid oxidase (glycolate oxidase) 1, Plasma, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Medication, General manager, Safety, Patient, Therapy, Profile, Society, Kidney, Chronic kidney disease, Erythema, HAO1, ESC, RNA interference, American Society of Nephrology, Skin, Heart, Pharmaceutical industry, Birth control

OXLUMO is indicated for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients.

Key Points: 
  • OXLUMO is indicated for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients.
  • The most common adverse reaction that occurred in patients treated with OXLUMO was injection site reaction (38%).
  • No data are available on the presence of OXLUMO in human milk or its effects on breastfed infants or milk production.
  • Lumasiran is a subcutaneously administered RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) in development for the treatment of advanced primary hyperoxaluria type 1 (PH1).

Alnylam Presents Positive Results from ILLUMINATE-C Phase 3 Study of Lumasiran in Patients with Advanced Primary Hyperoxaluria Type 1

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Vendredi, novembre 5, 2021
Research, Clinical Trials, Biotechnology, Other Health, Health, Pharmaceutical, General Health, Other Science, Science, GO, Society, Milk, Safety, Alnylam Pharmaceuticals, AES, American Society of Nephrology, Pregnancy, CKD, Cohort study, Plasma, Kidney, Therapeutic index, Baylor College of Medicine, AUC, General manager, Disease, RNA interference, Swelling, Department, ESC, EGFR, EMA, Food, Profile, European Medicines Agency, Associate professor, Chronic kidney disease, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, FDA, Medication, Dialysis, 1991 World Sportscar Championship, Pain, Itch, Hyperoxaluria, Therapy, Diagnosis, Severe cognitive impairment, Enhance, ASN, Hydroxyacid oxidase (glycolate oxidase) 1, PH1, Cardiomyopathy, Nasdaq, Patient, HAO1, Risk, AE, Bone, Child, Injection site reaction, Erythema, Adult, Pharmaceutical industry, Birth control, Lumasiran, ILLUMINATE-C Phase 3 Study, Primary Hyperoxaluria Type 1 (PH1), RNAi, Alnylam Pharmaceuticals, LUMASIRAN, ILLUMINATE-C PHASE 3 STUDY, PRIMARY HYPEROXALURIA TYPE 1 (PH1), RNAI, ALNYLAM PHARMACEUTICALS

PH1 patients progressing to or being diagnosed with end-stage kidney disease often have substantial elevations in plasma oxalate despite intensive hemodialysis.

Key Points: 
  • PH1 patients progressing to or being diagnosed with end-stage kidney disease often have substantial elevations in plasma oxalate despite intensive hemodialysis.
  • ILLUMINATE-C enrolled 21 patients: six patients in Cohort A who did not require dialysis and 15 patients on hemodialysis in Cohort B.
  • OXLUMO is indicated for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients.
  • Lumasiran is a subcutaneously administered RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) in development for the treatment of primary hyperoxaluria type 1 (PH1).