COL4A4

Alport Syndrome Market to Witness Upsurge in Growth at a Massive CAGR of 69% by 2034 | DelveInsight

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星期三, 四月 17, 2024
USD, Urine, ACE, Finerenone, ESRD, Kidney, Otorhinolaryngology, Nephrotoxicity, Mutation, Hearing, Diagnosis, Glomerular filtration rate, Hearing loss, Blood, Swelling, Proteinuria, Kidney failure, Cochlea, Alport syndrome, Nephrology, EU4, GFR, ADAS, Heredity, Research, Genetic counseling, Kidney disease, Novartis, ARAS, Ophthalmology, COL4A4, Patient, Kidney transplantation, LAS, Dialysis, DSM-IV codes, COL4A3, Quality of life, Genetic disorder, Gene, Pharmaceutical industry

LAS VEGAS, April 17, 2024 /PRNewswire/ -- DelveInsight's Alport Syndrome Market Insights report includes a comprehensive understanding of current treatment practices, Alport syndrome emerging drugs, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan].

Key Points: 
  • Leading Alport syndrome companies such as Eloxx Pharmaceuticals, Chinook Therapeutics (A Novartis company), Bayer, Calliditas Therapeutics, Evotec, and others are developing novel Alport syndrome drugs that can be available in the Alport syndrome market in the coming years.
  • The disorder manifests in different forms such as autosomal recessive Alport syndrome (ARAS), X-linked Alport syndrome (XLAS), and autosomal dominant Alport syndrome (ADAS).
  • The X-linked Alport syndrome (XLAS) is the most prevalent subtype of Alport syndrome with around 11K cases in 2023 in the US while autosomal dominant Alport syndrome (ADAS) is the least prevalent subtype of Alport syndrome.
  • To know more about Alport syndrome treatment guidelines, visit @ Alport Syndrome Management

Alport Syndrome Market to Witness Upsurge in Growth at a Massive CAGR of 69% by 2034 | DelveInsight

Retrieved on: 
星期三, 四月 17, 2024
USD, Urine, ACE, Finerenone, ESRD, Kidney, Otorhinolaryngology, Nephrotoxicity, Mutation, Hearing, Diagnosis, Glomerular filtration rate, Hearing loss, Blood, Swelling, Proteinuria, Kidney failure, Cochlea, Alport syndrome, Nephrology, EU4, GFR, ADAS, Heredity, Research, Genetic counseling, Kidney disease, Novartis, ARAS, Ophthalmology, COL4A4, Patient, Kidney transplantation, LAS, Dialysis, DSM-IV codes, COL4A3, Quality of life, Genetic disorder, Gene, Pharmaceutical industry

LAS VEGAS, April 17, 2024 /PRNewswire/ -- DelveInsight's Alport Syndrome Market Insights report includes a comprehensive understanding of current treatment practices, Alport syndrome emerging drugs, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan].

Key Points: 
  • Leading Alport syndrome companies such as Eloxx Pharmaceuticals, Chinook Therapeutics (A Novartis company), Bayer, Calliditas Therapeutics, Evotec, and others are developing novel Alport syndrome drugs that can be available in the Alport syndrome market in the coming years.
  • The disorder manifests in different forms such as autosomal recessive Alport syndrome (ARAS), X-linked Alport syndrome (XLAS), and autosomal dominant Alport syndrome (ADAS).
  • The X-linked Alport syndrome (XLAS) is the most prevalent subtype of Alport syndrome with around 11K cases in 2023 in the US while autosomal dominant Alport syndrome (ADAS) is the least prevalent subtype of Alport syndrome.
  • To know more about Alport syndrome treatment guidelines, visit @ Alport Syndrome Management

Eloxx Pharmaceuticals Reports Second Quarter 2023 Financial and Operating Results and Provides Business Update

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星期一, 八月 14, 2023
COL7, G&A, Cystic fibrosis, Congress, Investigational New Drug, Patient, R, Disease, MYC, Research, Concha bullosa, Alport syndrome, Proteinuria, Cystic Fibrosis Foundation, MAD, COL, Rare, FDA, IND, COL4A4, Collagen, Kidney failure, Skin, Natural history, European Renal Association, Glomerular basement membrane, Cancer, Ultrafiltration, Fibroblast, Colorectal cancer, Radar, Podocyte, Familial adenomatous polyposis, Clinical trial, SAD, FAP, Large intestine, RNA, Trial of the century, Keratinocyte, APC, Food, UPCR, Pharmaceutical industry, Vaccine, Dietary supplement, Health insurance, Alport, RDEB, Creatinine

Podocytes are specialized cells that bind to the glomerular basement membrane and form finger-like extensions called foot processes that enable efficient ultrafiltration.

Key Points: 
  • Podocytes are specialized cells that bind to the glomerular basement membrane and form finger-like extensions called foot processes that enable efficient ultrafiltration.
  • Podocyte injury leads to the effacement (loss) of podocyte foot processes and proteinuria in nearly all cases of Alport syndrome.
  • An IND application for ELX-02 is expected to be submitted to the FDA in the third quarter of 2023.
  • Eloxx remains focused on its liquidity position and is committed to raising additional capital in the near term in order to fund its operating plan through the end of 2023 and beyond.

Eloxx Pharmaceuticals Reports Drug Response in All Patients Treated with ELX-02 in Phase 2 Clinical Study for Alport Syndrome

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星期一, 八月 14, 2023
Glomerular basement membrane, Mutation, Ultrafiltration, TEM, RNA, Alport syndrome, Trial of the century, Proteinuria, Patient, Rare, Podocyte, COL4A4, Biopsy, Disease, Pharmaceutical industry, Alport

WATERTOWN, Mass., Aug. 14, 2023 (GLOBE NEWSWIRE) -- Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX), a leader in ribosomal RNA-targeted genetic therapies for rare diseases, today reported additional positive results from its proof-of-concept Phase 2 open-label clinical trial (NCT05448755) of ELX-02 for the treatment of Alport syndrome after eight weeks of treatment. Electron microscopy assessment of kidney biopsies demonstrated an improvement in foot process effacement in all three treated patients. These results are consistent with the disease modifying effect of restoration of COL4A4 protein with ELX-02 and with previously reported data from preclinical studies.

Key Points: 
  • Electron microscopy assessment of kidney biopsies demonstrated an improvement in foot process effacement in all three treated patients.
  • Podocyte injury leads to the effacement (loss) of podocyte foot processes and proteinuria in nearly all cases of Alport syndrome.
  • This is the first potential therapy for Alport syndrome that has shown an improvement of podocyte foot process effacement.
  • The improvement in podocyte foot process effacement in all three patients treated confirms the disease modifying potential of ELX-02.

Eloxx Pharmaceuticals Highlights Recent Alport Syndrome Natural History Data Presented at 60th ERA Congress

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星期三, 六月 21, 2023
Male, Kidney failure, Natural history, European Renal Association, Mutation, Research, RNA, Alport syndrome, ERA, Congress, COL4A5, HIV disease progression rates, Patient, Risk, National Register of Historic Places, Rare, Radar, COL4A4, Pharmaceutical industry, Alport

WATERTOWN, Mass., June 21, 2023 (GLOBE NEWSWIRE) -- Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX), a leader in ribosomal RNA-targeted genetic therapies for rare diseases, today highlighted recent Alport syndrome natural history data presented at the 60th ERA (European Renal Association) Congress.

Key Points: 
  • Data from RaDaR natural history study indicates that Alport syndrome patients with autosomal recessive COL4A4 mutations have severest disease, with a more rapid progression to kidney failure
    WATERTOWN, Mass., June 21, 2023 (GLOBE NEWSWIRE) -- Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX), a leader in ribosomal RNA-targeted genetic therapies for rare diseases, today highlighted recent Alport syndrome natural history data presented at the 60th ERA (European Renal Association) Congress.
  • Katie Wong, Clinical Research Fellow at University College London Department of Renal Medicine, presented “Alport Syndrome Natural History from the RaDaR Registry: Associations with gene, variant type and sex”, a natural history study that aims to describe demographics and investigate renal outcomes associated with pathogenic mutations in Alport syndrome patients.
  • One key finding indicates that approximately 11% of Alport syndrome patients have autosomal recessive COL4A4 mutations and have severest disease.
  • “We are grateful to the patients who participated in the natural history study, honored to participate in the research, and look forward to continuing our support of the Alport syndrome community.”

Eloxx Pharmaceuticals Reports Fourth Quarter 2022 Financial and Operating Results and Provides Business Update

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星期一, 四月 3, 2023
COL7, Polyp, Collagen, Skin, Drug development, Hearing loss, Familial adenomatous polyposis, Survival, Clinical trial, Mutation, American Society of Nephrology, G&A, Cystic fibrosis, FAP, Fibroblast, RNA, Alport syndrome, Trial of the century, ASN, Cancer, Proteinuria, COL4A5, Investigational New Drug, Genetic disorder, Patient, COL4A3, Keratinocyte, Kidney, Adenoma, Cystic Fibrosis Foundation, COL, Growth, Epidermolysis bullosa dystrophica, APC, Rare, R, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, IND, COL4A4, Society, Conference, Food, Kidney disease, Pharmaceutical industry, Cryptocurrency, RDEB, Alport

WATERTOWN, Mass., April 03, 2023 (GLOBE NEWSWIRE) -- Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX), a leader in ribosomal RNA-targeted genetic therapies for rare diseases, today reported its financial results for the three months ended December 31, 2022 and provided a business update.

Key Points: 
  • Alport syndrome is a rare genetic disorder characterized by kidney disease with high levels of proteinuria, hearing loss and eye abnormalities.
  • Eloxx presented a poster highlighting the activity of ELX-02 across a range of COL4A5 mutations in preclinical models at the American Society of Nephrology (ASN) Kidney Week 2022 Conference in early November 2022.
  • Recent preclinical results demonstrated read-through activity of ZKN-013 in multiple COL7 genotypes across multiple RDEB patient derived fibroblasts and keratinocytes.
  • R&D expenses were $3.3 million for the three months ended December 31, 2022, which includes $0.3 million in stock-based compensation.

Eloxx Pharmaceuticals Announces Opening of Clinical Trial Sites for Phase 2 Study of ELX-02 for the Treatment of Alport Syndrome

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星期二, 十一月 1, 2022
RNA transfection, RNA, Plasma, RMAS, Science, CF, Mutation, SEC, Clinical trial, Cancer, GLOBE, Patient, Royal commission, Research, Eukaryotic ribosome, COVID-19, Allele, Disease, COL4A3, File, European Commission, Degenerative disease, Cystic fibrosis, Proteinuria, Kidney disease, Safety, Genetic disorder, Patent, Judgement, COL4A5, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, NASDAQ, Private Securities Litigation Reform Act, Polycystic kidney disease, Hearing loss, FDA, Kidney, Food, Fast Track, Alport syndrome, Autosome, Forward-looking statement, Gene, COL4A4, Orphan, Pharmaceutical industry, Alport

Additional sites are expected to be opened and actively recruiting Alport patients in the coming weeks, said Sumit Aggarwal, President and Chief Executive Officer of Eloxx.

Key Points: 
  • Additional sites are expected to be opened and actively recruiting Alport patients in the coming weeks, said Sumit Aggarwal, President and Chief Executive Officer of Eloxx.
  • Alport patients with nonsense mutations have significantly worse clinical outcomes than other Alport patients and have no disease modifying treatment options.
  • This proof-of-concept clinical trial will include up to eight Alport syndrome patients with nonsense mutations in the second half of 2022.
  • Approximately 6% to 7% of Alport syndrome patients, or approximately 9,400 to 12,750 individuals, are estimated to have nonsense mutations.

Eloxx Pharmaceuticals Announces New Development Program for ELX-02 for the Treatment of Alport Syndrome

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星期三, 三月 30, 2022
Disease, Autosome, Private Securities Litigation Reform Act, COL4A4, Plasma, Population, NASDAQ, Degenerative disease, COL4A3, Patent, Polycystic kidney disease, Eukaryotic ribosome, Forward-looking statement, Clinical trial, Kidney, Hearing loss, Mutation, Judgement, Proteinuria, SEC, Research, Program, Alport syndrome, Patient, Cancer, Kidney disease, Allele, RNA, Cystic fibrosis, Safety, RNA transfection, COVID-19, GLOBE, Gene, Genetic disorder, Science, RMAS, File, Pharmaceutical industry, COL4A5, Alport

WATERTOWN, Mass., March 30, 2022 (GLOBE NEWSWIRE) -- Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX), a leader in ribosomal RNA-targeted genetic therapies for rare diseases, today announced it has expanded the ELX-02 development program to include the treatment of Alport syndrome, a rare kidney disease. Clinical testing of ELX-02 in Alport syndrome is expected to initiate in the second half of 2022, with topline results expected in the first half of 2023.

Key Points: 
  • Clinical testing of ELX-02 in Alport syndrome is expected to initiate in the second half of 2022, with topline results expected in the first half of 2023.
  • Approximately 6% to 7% of Alport syndrome patients, or approximately 9,400 to 12,750 individuals, are estimated to have nonsense mutations.
  • These patients have significantly worse clinical outcomes than other Alport patients and have no disease modifying treatment options.
  • Eloxx intends to initiate a proof-of-concept clinical trial in up to eight Alport syndrome patients with nonsense mutations in the second half of 2022.