PKD2

Mironid extends Series A round raising £35 million to date for development of first-in-class small molecules to treat life-threatening hereditary kidney disease

Retrieved on: 
Wednesday, September 13, 2023
European Investment Fund, Camp, Kidney failure, Drug development, University, Therapy, AMP, Polyuria, Sofinnova, Cell proliferation, Autosomal dominant polycystic kidney disease, PKD2, Cyst, Patient, University of Strathclyde, PKD1, Series, AICA ribonucleotide, Kidney, Growth, ADPKD, Scottish Enterprise, IND, Disease, Pharmaceutical industry

The Company will use the proceeds to progress its lead discovery programme through IND-enabling studies and to extend its patent estate.

Key Points: 
  • The Company will use the proceeds to progress its lead discovery programme through IND-enabling studies and to extend its patent estate.
  • It affects over 12 million people worldwide, with 50% of patients developing kidney failure by the age of 60.
  • The disease is caused predominantly by mutations in the PKD1 or PKD2 gene and is characterized by uncontrolled growth of fluid-filled cysts in the kidney.
  • Robust in vitro and disease model data show significant efficacy across all disease endpoints, including a reduction in cyst number and kidney volume.

Global Autosomal Dominant Polycystic Kidney Disease Epidemiology Forecast Report to 2032 - ResearchAndMarkets.com

Retrieved on: 
Friday, October 7, 2022
Genetics, Pharmaceutical, Health, Arachnoid, Epidemiology, Cyst, Hypertension, Colin L. Masters, Doctor of Philosophy, ERM, PKD2, Growth, Population, Diagnosis, Mitral valve prolapse, Kidney failure, Patient, Urinary tract infection, Degenerative disease, Renal cysts and diabetes syndrome, Kidney, Diverticular disease, Polycystic kidney disease, Hernia, Stabat Mater, Hematuria, Adult, Liver, United Kingdom, Fibrosis, PKD1, Master's degree, Intracranial aneurysm, Autosome, Left ventricular hypertrophy, Chronic kidney disease, Pancreas, Autosomal dominant polycystic kidney disease, PKD, Vaccine, Pharmaceutical industry, Lithium, Medical device, Health insurance, Management

The "Autosomal Dominant Polycystic Kidney Disease - Epidemiology Forecast - 2032" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Autosomal Dominant Polycystic Kidney Disease - Epidemiology Forecast - 2032" report has been added to ResearchAndMarkets.com's offering.
  • This report delivers an in-depth understanding of Autosomal Dominant Polycystic Kidney Disease, historical and forecasted epidemiology as well as Autosomal Dominant Polycystic Kidney Disease trends in the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom) and Japan.
  • The diagnosed prevalent cases of Autosomal Dominant Polycystic Kidney Disease are likely to change by 2032 in the forecast period 2022-2032.
  • The report provides the segmentation of the disease epidemiology for the 7MM, a total number of total Diagnosed Prevalent Cases of Autosomal Dominant Polycystic Kidney Disease, Age-specific cases of Autosomal Dominant Polycystic Kidney Disease, and Mutation-specific cases of Autosomal Dominant Polycystic Kidney Disease.

Autosomal Dominant Polycystic Kidney Disease Market to Surge at a Significant CAGR of 14.5% by 2032 | DelveInsight

Retrieved on: 
Monday, September 19, 2022
Adult, Pediatrics, PKD1, Urology, Research, PKD, Patient, Physician, United Kingdom, PKD2, Kidney, Disease, Mario Negri Institute for Pharmacological Research, AL, Diet, Pharmacological Research, Kadmon Corporation, Prevalence, Back pain, Pain, Polycystic kidney disease, Time-invariant system, Risk, Population, Autosome, Tolvaptan, Otsuka Pharmaceutical, Water, Reata Pharmaceuticals, HIV disease progression rates, Regulus Therapeutics, Ryukyu Kingdom, Cyst, LAS, Genetic counseling, Nephrology, LAR, Headache, Therapy, Galapagos NV, Pharmaceutical industry, Lithium, Tesevatinib, ADPKD, Bardoxolone methyl

LAS VEGAS, Sept. 19, 2022 /PRNewswire/ -- DelveInsight's ADPKD Market Insights report includes a comprehensive understanding of current treatment practices, ADPKD emerging drugs, market share of individual therapies, and current and forecasted market size from 2019 to 2032, segmented into 7MM [the United States, the EU5 (the United Kingdom, Italy, Spain, France, and Germany), Japan].

Key Points: 
  • In addition, the approval and entry of new drugs will change the ADPKD market dynamics in the future.
  • As per DelveInsight estimates, the ADPKDmarket size in the 7MM was approximately USD 1,077 million in 2021.
  • Autosomal dominant polycystic kidney disease also called "adult PKD," is the most common inherited kidney disorder characterized by cyst growth in the kidneys, which increases with disease progression and results in renal failure.
  • Implementing these measures early in the disease should slow the progression of kidney (renal) disease and, to some extent, preserve kidney function.

Regulus Therapeutics Announces Receipt of FDA Orphan Drug Designation for RGLS8429 for the Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Retrieved on: 
Tuesday, June 21, 2022
Risk factors for tuberculosis, Chronic kidney disease, Security (finance), FDA, PKD2, Drug development, COVID-19, Therapy, Risk, Food and Drug Administration Amendments Act of 2007, Safety, Degenerative disease, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Kidney, CEO, Transplant, Liver, Company, U.S. Securities and Exchange Commission, Pharmacokinetics, Private Securities Litigation Reform Act, ODD, MicroRNA, Disease, Drug discovery, ADPKD, Form 10-Q, CA, PKD1, Dialysis, Toxicology, Food, Regulus Therapeutics, Patient, Cyst, Diagnosis, Multimedia, SAN, Autosomal dominant polycystic kidney disease, Pharmaceutical industry, Regulus

(Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the "Company" or "Regulus"), today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to RGLS8429 for the treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD).

Key Points: 
  • (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the "Company" or "Regulus"), today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to RGLS8429 for the treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD).
  • Autosomal Dominant Polycystic Kidney Disease (ADPKD), caused by mutations in the PKD1 or PKD2 genes, is among the most common human monogenic disorders and a leading cause of end-stage renal disease.
  • RGLS8429 is a novel, next generation oligonucleotide for the treatment of ADPKD designed to inhibit miR-17 and to preferentially target the kidney.
  • Regulus Therapeutics Inc.(Nasdaq: RGLS) is a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs.

Regulus Therapeutics Announces FDA Acceptance of Investigational New Drug (IND) Application for RGLS8429 for the Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Retrieved on: 
Wednesday, May 11, 2022
Regulus Therapeutics, COVID-19, Drug discovery, Private Securities Litigation Reform Act, IND, Autosomal dominant polycystic kidney disease, Pharmacokinetics, Cyst, Company, Kidney, Food, Safety, PKD1, Toxicology, CA, Patient, MAD, Trial of the century, Autosome, Program, Risk, MicroRNA, FDA, PKD2, Therapy, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Multimedia, Adult, ADPKD, Disease, Polycystic kidney disease, Liver, U.S. Securities and Exchange Commission, Food and Drug Administration Amendments Act of 2007, SAD, SAN, Chronic kidney disease, Pharmaceutical industry, Regulus

SAN DIEGO, May 11, 2022 /PRNewswire/ -- Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the "Company" or "Regulus"), today announced that the U.S. Food and Drug Administration (FDA) has accepted its Investigational New Drug (IND) Application for RGLS8429 for the treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD), enabling the Company to initiate its planned Phase 1 clinical study of RGSL8429 in healthy volunteers.

Key Points: 
  • The Company will conduct a Phase 1 single-ascending dose (SAD) study in healthy volunteers to assess safety, tolerability and pharmacokinetics of RGLS8429.
  • Excessive kidney cyst cell proliferation, a central pathological feature, ultimately leads to end-stage renal disease in approximately 50% of ADPKD patients by age 60.
  • RGLS8429 is a novel, next generation oligonucleotide designed to inhibit miR-17 and to preferentially target the kidney.
  • Regulus undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.