COL7

Almirall and Eloxx Pharmaceuticals Enter into Exclusive Agreement to license ZKN-013 for rare dermatological diseases

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水曜日, 3月 13, 2024
COL7, FAP, Cancer, Patient, APC, Mouse, ALM, Ribosome, BME, Gastrointestinal disease, OTC, Skin, Polyp (medicine), Therapy, Kidney, Pharmaceutical industry

BARCELONA, Spain and WATERTOWN, Mass., March 13, 2024 (GLOBE NEWSWIRE) -- Almirall, S.A. (BME:ALM) and Eloxx Pharmaceuticals, Inc. (OTC: ELOX) announced today that the companies have entered into an exclusive license agreement for the asset ZKN-013.

Key Points: 
  • BARCELONA, Spain and WATERTOWN, Mass., March 13, 2024 (GLOBE NEWSWIRE) -- Almirall, S.A. (BME:ALM) and Eloxx Pharmaceuticals, Inc. (OTC: ELOX) announced today that the companies have entered into an exclusive license agreement for the asset ZKN-013.
  • Under the agreement, Almirall obtains global rights to develop and commercialize ZKN-013, including for the use in orphan dermatological diseases.
  • ZKN-013 is a potentially promising oral, nonsense mutation readthrough drug, which enables the host cells to produce functional proteins which counteract the root cause of these rare dermatological and potentially other diseases.
  • The drug candidate is expected to shortly enter into Phase I development in healthy volunteers.

Almirall and Eloxx Pharmaceuticals Enter into Exclusive Agreement to License ZKN-013 for Rare Dermatological Diseases

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水曜日, 3月 13, 2024
Science, Other Science, Biotechnology, Research, Pharmaceutical, Health, Clinical Trials, Other Health, COL7, FAP, Cancer, Patient, APC, Mouse, ALM, Ribosome, BME, Gastrointestinal disease, OTC, Skin, Polyp (medicine), Therapy, Kidney, Pharmaceutical industry

Almirall, S.A. (BME:ALM) and Eloxx Pharmaceuticals, Inc. (OTC: ELOX) announced today that the companies have entered into an exclusive license agreement for the asset ZKN-013.

Key Points: 
  • Almirall, S.A. (BME:ALM) and Eloxx Pharmaceuticals, Inc. (OTC: ELOX) announced today that the companies have entered into an exclusive license agreement for the asset ZKN-013.
  • Under the agreement, Almirall obtains global rights to develop and commercialize ZKN-013, including for the use in orphan dermatological diseases.
  • ZKN-013 is a potentially promising oral, nonsense mutation readthrough drug, which enables the host cells to produce functional proteins which counteract the root cause of these rare dermatological and potentially other diseases.
  • The drug candidate is expected to shortly enter into Phase I development in healthy volunteers.

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

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月曜日, 8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.

FDA Approves First Topical Gene Therapy for Treatment of Wounds in Patients with Dystrophic Epidermolysis Bullosa

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金曜日, 5月 19, 2023
COL7, COL7A1, DEB, Dermis, Chills, RDEB, Connective tissue, Food, Center for Biologics Evaluation and Research, Epidermis, Nail, Concha bullosa, Cough, Trial of the century, Rash, Hydrogen peroxide, VII, Genetic disorder, Patient, Paratriathlon, Research, FDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Red, Skin, Caregiver, Safety, Wound healing, Vaccine, Birth control, Nursing

This gene encodes type VII collagen (COL7), which is an essential protein that helps strengthen and stabilize the outer and middle layers of the skin.

Key Points: 
  • This gene encodes type VII collagen (COL7), which is an essential protein that helps strengthen and stabilize the outer and middle layers of the skin.
  • When COL7A1 is deficient, skin layers can separate, causing painful and debilitating blisters and wounds.
  • DEB usually presents itself at birth and is divided into two major types depending on the inheritance pattern: recessive dystrophic epidermolysis bullosa (RDEB) and dominant dystrophic epidermolysis bullosa (DDEB).
  • Vyjuvek is a genetically modified (engineered in a laboratory) herpes-simplex virus used to deliver normal copies of the COL7A1 gene to the wounds.

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

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月曜日, 5月 15, 2023
COL7, Polyp, Skin, Drug development, Biopsy, Hearing loss, Familial adenomatous polyposis, Survival, Clinical trial, Lead, SAD, G&A, Cystic fibrosis, FAP, Fibroblast, RNA, Alport syndrome, Cancer, Kidney disease, Proteinuria, Genetic disorder, Patient, Keratinocyte, Cystic Fibrosis Foundation, Adenoma, COL, Growth, MAD, APC, Rare, R, FDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Food, Pharmaceutical industry, Vaccine, RDEB, Alport

WATERTOWN, Mass., May 15, 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 March 31, 2023 and provided a business update.

Key Points: 
  • Eloxx has dosed three patients with ELX-02 in the ongoing proof-of-concept Phase 2 open-label clinical trial (NCT05448755).
  • Recent preclinical results demonstrated read-through activity of ZKN-013 in multiple COL7 genotypes across multiple RDEB patient derived fibroblasts and keratinocytes.
  • In January 2023, Eloxx published positive results from a study in the APCMin (multiple intestinal neoplasia) model evaluating the potential of ZKN-013 to treat FAP.
  • In March 2023, we amended the terms of the Hercules Term Loan Agreement and repaid $7.5 million of the outstanding principal.

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

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月曜日, 4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 Submission of Investigational New Drug (IND) Application for ZKN-013

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火曜日, 3月 28, 2023
COL7, Polyp, Polyp (medicine), Skin, Drug development, Familial adenomatous polyposis, Survival, Mutation, FAP, Fibroblast, RNA, Cancer, Investigational New Drug, JEB, Patient, Keratinocyte, DSM-IV codes, Adenoma, COL, Growth, Epidermolysis bullosa dystrophica, APC, Rare, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, IND, Food, Vaccine, Pharmaceutical industry, RDEB

WATERTOWN, Mass., March 28, 2023 (GLOBE NEWSWIRE) -- Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX), a leader in ribosomal RNA-targeted genetic therapies for rare diseases, today announced the submission of an Investigational New Drug (IND) application with the U.S. Food and Drug Administration for ZKN-013 for the treatment of recessive Dystrophic Epidermolysis Bullosa (RDEB) with nonsense mutations. RDEB is a rare skin disease characterized by mutations in Collagen7 gene.

Key Points: 
  • “This IND application is an important milestone towards providing a treatment option for patients with RDEB and JEB, as there are currently no approved disease-modifying treatments,” said Sumit Aggarwal, President and Chief Executive Officer of Eloxx.
  • FAP, a rare inherited disease with no approved drug therapies, is characterized by proliferation of colon polyps.
  • Eloxx is targeting a subset of patients that have nonsense mutations in the Adenomatous Polyposis Coli (APC) gene that is truncated in these patients.
  • In January 2023, Eloxx published positive results from a study in the APCMin (multiple intestinal neoplasia) model evaluating the potential of ZKN-013 to treat FAP.

Dystrophic Epidermolysis Bullosa Drug Pipeline Analysis Report 2022: Comprehensive Insights About 15+ Companies and 15+ Pipeline Drugs - ResearchAndMarkets.com

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月曜日, 12月 19, 2022
Science, Other Science, Biotechnology, Research, Pharmaceutical, Health, Genetics, Other Health, Epidermolysis bullosa dystrophica, VII, II, Patient, RDEB, Tissue, Shire (pharmaceutical company), Disease, III, Concha bullosa, Discovery, Clinical trial, News, COL7, Wound healing, Therapy, NDA, Survival, DEB, Skin, Research, Tooth discoloration, Acquisition, Medical device, Medical imaging, Pharmaceutical industry

This report provides comprehensive insights about 15+ companies and 15+ pipeline drugs in Dystrophic Epidermolysis Bullosa pipeline landscape.

Key Points: 
  • This report provides comprehensive insights about 15+ companies and 15+ pipeline drugs in Dystrophic Epidermolysis Bullosa pipeline landscape.
  • The assessment part of the report embraces, in depth Dystrophic Epidermolysis Bullosa commercial assessment and clinical assessment of the pipeline products under development.
  • This segment of the Dystrophic Epidermolysis Bullosa report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery.
  • Our in-depth analysis of the pipeline assets (in early-stage, mid-stage and late stage of development for the treatment of Dystrophic Epidermolysis Bullosa) includes therapeutic assessment and comparative analysis.

New England Journal of Medicine Publishes Phase 3 Data on B-VEC in Patients with Dystrophic Epidermolysis Bullosa

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水曜日, 12月 14, 2022
Associate, Priority review, Marketing, Priority, Epidermolysis bullosa dystrophica, Finger, Tears, Trauma, Fibrosis, NEJM, Dermis, Wound healing, Stanford University Medical Center, EMA, MA, Squamous cell carcinoma, Concha bullosa, Food, FDA, US Foods, The, Safety, COL7A1, DEB, Blister, Skin, Research and development, Manuscript, Degenerative disease, Securities and Exchange Commission (Philippines), Suma, The New England Journal of Medicine, HSV, Krystal, Hope, Disease, Stanford University School of Medicine, Tissue, Patient, VII, BLA, Prescription Drug User Fee Act, COL7, Food and Drug Administration, KRYS, Risk, Epidermis, Pharmaceutical industry, Vaccine, Dermatology

The full manuscript, titled “Trial of Beremagene Geperpavec (B-VEC) for Dystrophic Epidermolysis Bullosa,” will appear in the December 15, 2022 issue of the NEJM.

Key Points: 
  • The full manuscript, titled “Trial of Beremagene Geperpavec (B-VEC) for Dystrophic Epidermolysis Bullosa,” will appear in the December 15, 2022 issue of the NEJM.
  • The GEM-3 trial was a randomized, double-blind, intra-patient placebo-controlled multi-center trial designed to evaluate the efficacy and safety of B-VEC for the treatment of DEB.
  • In the trial, matched wounds receiving topical B-VEC or placebo were evaluated in 31 DEB patients over 26 weeks.
  • Krystal Biotech, Inc. (NASDAQ: KRYS) is a biotechnology company focused on developing and commercializing genetic medicines for patients with rare diseases.

Krystal Biotech Receives Positive Opinion from EMA Pediatric Committee on the Pediatric Investigation Plan for B-VEC for the treatment of Dystrophic Epidermolysis Bullosa

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水曜日, 9月 21, 2022
Priority, Tears, Fibrosis, Twitter, Risk, PRV, FDA, Food, Finger, VII, European Medicines Agency, The, Epidermis, LinkedIn, Company, Population, Suma, Dermis, Degenerative disease, Patient, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Trauma, Securities and Exchange Commission (Philippines), COL7, PIP, Blister, DEB, MAA, Tissue, Private Securities Litigation Reform Act, Skin, NASDAQ, Squamous cell carcinoma, Lung, KRYS, Priority review, GLOBE, Marketing, EMA, Research and development, COL7A1, Pharmaceutical industry, EU

PITTSBURGH, Sept. 21, 2022 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), the leader in redosable gene therapy, announced today that the European Medicines Agency (EMA) Pediatric Committee (PDCO) has adopted a positive opinion on the Pediatric Investigation Plan (PIP) for beremagene geperpavec (B-VEC) for the treatment of dystrophic epidermolysis bullosa.

Key Points: 
  • PITTSBURGH, Sept. 21, 2022 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the Company) (NASDAQ: KRYS), the leader in redosable gene therapy, announced today that the European Medicines Agency (EMA) Pediatric Committee (PDCO) has adopted a positive opinion on the Pediatric Investigation Plan (PIP) for beremagene geperpavec (B-VEC) for the treatment of dystrophic epidermolysis bullosa.
  • The positive opinion is based on the B-VEC non-clinical safety program as well as data from the clinical studies conducted in the US that will be included in the upcoming MAA.
  • The U.S. Food and Drug Administration (FDA) and EMA have each granted B-VEC orphan drug designation for the treatment of DEB.
  • The FDA has granted B-VEC fast track designation, Regenerative Medicine Advanced Therapy (RMAT) and rare pediatric disease designation for the treatment of DEB.