Systemic scleroderma

AnaMar Announces US and EU Orphan Drug Designation for AM1476 for Treating Systemic Sclerosis

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星期一, 二月 5, 2024
Inflammation, Systemic scleroderma, Biomarker, Patient, Diagnosis, FDA, EMA, Marketing, Scleroderma, Interstitial lung disease, Skin, Pulmonary fibrosis, Disability, Disease, Fibrosis, European Medicines Agency, Death, ODD, Autoimmune disease, ILD, Organ dysfunction, Pharmaceutical industry

AM1476 offers a unique dual-action approach to treat both skin and lung manifestations of systemic sclerosis.

Key Points: 
  • AM1476 offers a unique dual-action approach to treat both skin and lung manifestations of systemic sclerosis.
  • Systemic sclerosis is a chronic, progressive, autoimmune disease characterized by inflammation and fibrosis, i.e.
  • AnaMar's Chief Executive Officer, Dr. Ulf Ljungberg, said: “We are delighted with the FDA’s and EMA’s decisions to grant orphan drug designation to AM1476 for SSc.
  • Orphan drug designation provides certain benefits, including the potential for extensive marketing exclusivity following regulatory approval, reduction in regulatory fees and, in the case of EU, a centralized approval process.

aTyr Pharma to Present Posters Highlighting Importance of Neuropilin-2 in Immune Regulation at Keystone Symposia on Myeloid Cell Diversity

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星期一, 一月 29, 2024
Patient, Medicine, Fibrosis, Faculty, Scar, SAN, NRP2, Scleroderma, University, Small RNA, Disease, Neuropilin, Nebraska Medicine, ATyr Pharma, Inflammation, Systemic scleroderma, Sarcoidosis, Myelocyte, Immune system, Pharmaceutical industry

SAN DIEGO, Jan. 29, 2024 (GLOBE NEWSWIRE) -- aTyr Pharma, Inc. (Nasdaq: LIFE), a clinical stage biotechnology company engaged in the discovery and development of first-in-class medicines from its proprietary tRNA synthetase platform, today announced that the company will present two posters at the Keystone Symposia on Myeloid Cell Diversity: From Fundamental Biology to Disease States, which is being held January 28 – 31, 2024, in Banff, Alberta, Canada.

Key Points: 
  • Findings further demonstrate that efzofitimod modulates myeloid cells via the neuropilin-2 (NRP2) receptor to promote a unique anti-inflammatory mechanism.
  • Role of NRP2 in immune system validated by activity of NRP2 blocking antibody in preclinical models.
  • aTyr Pharma, San Diego; Faculty of Health and Medical Sciences, Copenhagen, Denmark.
  • These forms of ILD have limited therapeutic options and there is a need for safer and more effective, disease-modifying treatments that improve outcomes.

aTyr Pharma Announces Howard University President Emeritus Dr. Wayne A. I. Frederick as Advisor

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星期四, 一月 18, 2024
Mortality, ILD, Patient, Outline of health sciences, Wayne A. I. Frederick, Sarcoidosis, Research, Biology, Fibrosis, Associate, Scar, ACS, Dean (education), Howard University, SAN, Knowledge, University, National academy, Humana, Scleroderma, Business, Small RNA, American Surgical Association, Human, Disease, American College, Neuropilin, ATyr Pharma, Health, Inflammation, Organization, MD Anderson Cancer Center, Systemic scleroderma, College, Howard University Hospital, Abstract, Myelocyte, American Cancer Society, Pharmaceutical industry

Dr. Frederick is President Emeritus of Howard University, having served as President from 2014 to 2023.

Key Points: 
  • Dr. Frederick is President Emeritus of Howard University, having served as President from 2014 to 2023.
  • “We are honored to welcome a distinguished physician executive such as Dr. Frederick as an advisor to aTyr,” said Sanjay S. Shukla, M.D., M.S., President and Chief Executive Officer of aTyr.
  • He is also the distinguished Charles R. Drew Professor of Surgery at the Howard University College of Medicine and a practicing cancer surgeon at Howard University Hospital.
  • Dr. Frederick earned a B.S., M.D., and completed his surgical residency training at Howard University Hospital.

Cabaletta Bio Receives Additional FDA Fast Track Designations for CABA-201 in Dermatomyositis and Systemic Sclerosis

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星期一, 一月 8, 2024
Dermatomyositis, Food, GMG, IND, Research, FDA, SLE, Organ dysfunction, Scleroderma, Investigational New Drug, Systemic scleroderma, Myositis, Patient, Lupus nephritis, Pharmaceutical industry

PHILADELPHIA, Jan. 08, 2024 (GLOBE NEWSWIRE) -- Cabaletta Bio, Inc. (Nasdaq: CABA), a clinical-stage biotechnology company focused on developing and launching the first curative targeted cell therapies for patients with autoimmune diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted separate Fast Track Designations to CABA-201, an investigational 4-1BB-containing fully human CD19-CAR T cell therapy, for the treatment of patients with dermatomyositis to improve disease activity and for the treatment of patients with systemic sclerosis (SSc) to improve associated organ dysfunction.

Key Points: 
  • “The additional Fast Track Designations for CABA-201 in both dermatomyositis and systemic sclerosis, the second and third Fast Track Designations for CABA-201, provide the opportunity for expedited development and review of CABA-201 for the treatment of these autoimmune indications where there is a significant unmet need, despite currently available therapies,” said David J. Chang, M.D., Chief Medical Officer of Cabaletta.
  • “We believe these designations potentially accelerate our ability to launch the first targeted, and potentially curative, cell therapy for autoimmune diseases driven by B cells.
  • To date, Cabaletta has received clearance from the FDA for Investigational New Drug (IND) applications for CABA-201 in multiple autoimmune conditions including systemic lupus erythematosus (SLE), myositis, SSc and generalized myasthenia gravis (gMG).
  • Cabaletta is conducting four Phase 1/2 clinical trials with a total of nine cohorts that can advance simultaneously, employing a similar parallel cohort design and starting dose of 1 x 106 cells/kg without a dose escalation requirement.

aTyr Pharma Presents Poster Demonstrating Preclinical Effects of Efzofitimod in Rheumatoid Arthritis and Rheumatoid Arthritis-Associated ILD at the ACR Convergence 2023

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星期一, 十一月 13, 2023
NRP2, SAN, Neuropilin, Inflammation, Scar, Sarcoidosis, ILD, Scleroderma, Systemic scleroderma, Patient, Myelocyte, Rheumatoid arthritis, American College of Rheumatology, ATyr Pharma, American College, Small RNA, ACR, Poster, Fibrosis, Pharmaceutical industry

The poster will be available on the aTyr website once presented.

Key Points: 
  • The poster will be available on the aTyr website once presented.
  • The poster presents findings from preclinical models of rheumatoid arthritis (RA) and RA-associated ILD (RA-ILD), where NRP2, efzofitimod’s binding partner, is known to be expressed on pro-inflammatory synovial macrophages.
  • An animal knockout model for NRP2 demonstrated that NRP2 deficiency exacerbated disease pathology in preclinical models of inflammatory disease.
  • These forms of ILD have limited therapeutic options and there is a need for safer and more effective, disease-modifying treatments that improve outcomes.

aTyr Pharma Announces Third Quarter 2023 Results and Provides Corporate Update

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星期四, 十一月 9, 2023
NRP2, Research, Safety, SAN, Neuropilin, PST, D, BLA, Sarcoidosis, ILD, Scleroderma, International, Investment, Systemic scleroderma, ERS, Clinical trial, Patient, European Respiratory Society, DSMB, Rheumatoid arthritis, ATyr Pharma, American College, American College of Rheumatology, Small RNA, The Journal, Frontiers, Skin, ACR, Poster, Biologics license application, Pharmaceutical industry, Pharmacology, Kyorin

SAN DIEGO, Nov. 09, 2023 (GLOBE NEWSWIRE) -- aTyr Pharma, Inc. (Nasdaq: LIFE) (“aTyr” or the “Company”), a clinical stage biotechnology company engaged in the discovery and development of first-in-class medicines from its proprietary tRNA synthetase platform, today announced third quarter 2023 results and provided a corporate update.

Key Points: 
  • Ended the third quarter of 2023 with $105.6 million in cash, cash equivalents and investments.
  • SAN DIEGO, Nov. 09, 2023 (GLOBE NEWSWIRE) -- aTyr Pharma, Inc. (Nasdaq: LIFE) (“aTyr” or the “Company”), a clinical stage biotechnology company engaged in the discovery and development of first-in-class medicines from its proprietary tRNA synthetase platform, today announced third quarter 2023 results and provided a corporate update.
  • Based on current enrollment projections, the Company expects to complete enrollment in the study early in the second quarter of 2024.
  • G&A Expenses: General and administrative expenses were $2.6 million for the third quarter of 2023.

Global CD19 Antibody Market and Clinical Pipeline Outlook 2023-2028: A $10 Billion+ Market by 2028 - ResearchAndMarkets.com

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星期三, 十一月 22, 2023
Health, Pharmaceutical, Research, Antibody, Immune system, Non-Hodgkin lymphoma, Therapy, Lymphoid leukemia, Loncastuximab tesirine, Scleroderma, CRS, JW, IND, Tafasitamab, Tisagenlecleucel, B-cell lymphoma, Patient, Systemic scleroderma, CD19, Blinatumomab, FDA, Lupus, Acute leukemia, SLE, Chronic lymphocytic leukemia, Novartis, Sjögren syndrome, Neurotoxicity, Quality of life, NHL, Fast Track, ALL, Cytokine release syndrome, CLL, Vaccine, Pharmaceutical industry, Amgen

The "Global CD19 Antibody Market and Clinical Pipeline Outlook 2028" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Global CD19 Antibody Market and Clinical Pipeline Outlook 2028" report has been added to ResearchAndMarkets.com's offering.
  • This can be attributed to the rapid regulatory approvals, and the current pipeline of investigational CD19-targeting therapies shows potential for a further surge of this market cap.
  • CD19-targeting therapies have made a significant impact on the clinical landscape of cancer treatment.
  • Global CD19 Antibody Market and Clinical Pipeline Outlook 2028 Report Highlights:
    Global CD19 Antibody Market Opportunity: > USD 10 Billion By 2028
    Commercially Approved CD 19 Antibodies: 10 Antibodies
    Comprehensive Insights On CD19 Antibodies In Clinical Trials: > 190 Antibodies
    Global CD19 Antibodies Clinical Trials By Company, Indication and Phase

Chemomab Presentation at ACR Convergence 2023 Provides Further Support for Key Role of Its CCL24 Target in the Pathogenesis of Systemic Sclerosis

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星期四, 十一月 16, 2023
Fibroblast, Parallel Walk Test, Endothelium, Therapy, Pathology, Scleroderma, Systemic scleroderma, CMMB, Axis, Risk, Pulmonary hypertension, Patient, Nature, American College of Rheumatology, Autoimmune disease, Orthostatic hypertension, American College, CCR3, Malignancy, CCL24, Sclerosis, Communication, Doctor of Philosophy, PAH, Skin, ACR, TEL, Pharmaceutical industry, Disease

TEL AVIV, Israel, Nov. 16, 2023 /PRNewswire/ -- Chemomab Therapeutics Ltd. (Nasdaq: CMMB) (Chemomab), a clinical stage biotechnology company focused on the discovery and development of innovative therapeutics for fibro-inflammatory diseases with high unmet need, today reported on its poster presentation at the American College of Rheumatology (ACR) Convergence 2023 conference. The study, which was conducted by Chemomab researchers working in collaboration with academic scientists, analyzed serum samples and clinical data from patients with systemic sclerosis (SSc) to assess the effect of the soluble protein CCL24 on the pathogenesis of SSc and its association with key aspects of SSc pathology. Chemomab's first-in-class monoclonal antibody, CM-101, is designed to neutralize CCL24 and normalize CCL24-driven fibro-inflammatory disease processes. CM-101 has been studied extensively in preclinical and patient models of SSc. Chemomab has an open IND in the U.S. for a Phase 2 trial of CM-101 in systemic sclerosis patients.

Key Points: 
  • Chemomab's first-in-class monoclonal antibody, CM-101, is designed to neutralize CCL24 and normalize CCL24-driven fibro-inflammatory disease processes.
  • Chemomab has an open IND in the U.S. for a Phase 2 trial of CM-101 in systemic sclerosis patients.
  • Systemic sclerosis is an autoimmune disease characterized by vascular injury and extensive tissue fibrosis of the skin and internal organs.
  • Using cell-based assays, the researchers showed that CCL24, alongside factors present in the SSc microenvironment, enhances the EndMT process.

HSS Presents New Reproductive Health Research at the ACR Convergence 2023

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星期二, 十一月 14, 2023
Knowledge, Woman, Cervical cancer, Contraindication, Patient, Rheumatoid arthritis, Documentation, SLE, Infertility, Diagnosis, Rheumatology, Rheumatism, Fertility, Prevalence, Menopause, Vaccination, Cryopreservation, Flare, DSM-IV codes, Birth, Connective tissue, Hospital for Special Surgery, Doctor of Public Health, Female, Fetus, Smoking, Senior counsel, RD, SAN, Menstrual cycle, Weill Cornell Medicine, Immunosuppression, Risk, History, Hysterectomy, Inflammatory arthritis, Lupus, HSS, PAP, Fertilisation, OOC, Research, Human papillomavirus infection, Ageing, Scleroderma, Systemic scleroderma, Vaccine, American College, American College of Rheumatology, COVID-19, Fertility preservation, MPH, ACR, HPV, Hospital, Birth control, Nursing, Food delivery, MD

Additional research is planned to assess whether demographics, lifestyle and medical conditions may influence this potential association.

Key Points: 
  • Additional research is planned to assess whether demographics, lifestyle and medical conditions may influence this potential association.
  • This study surveyed women of reproductive age with systemic rheumatic disease about their use of contraceptives.
  • Data were collected on 249 women with reproductive capacity, including 147 with inflammatory arthritis and 102 with lupus-like disorders.
  • This study used data from the electronic health record of patients diagnosed with a rheumatic disease in adolescence, seen at HSS, to look at factors associated with HPV vaccination.

Cabaletta Bio Reports Third Quarter 2023 Financial Results and Provides Business Update

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星期四, 十一月 9, 2023
SLE, GMG, FDA, Systemic scleroderma, Research, CAR, CART, Report, American College, IND, Syndrome, Cyclophosphamide, MPV, MuSK protein, Dermatomyositis, Food, Nidec ASI, Myositis, Oxford, CD19, Molecular Therapy, Pemphigus, Good manufacturing practice, MG, Lupus, Conference, University, DM, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Maintenance, American College of Rheumatology, Development, Wuxi, San Diego Convention Center, Disease, Scleroderma, Immunoglobulin therapy, GMP, Autoimmunity, Investment, Patient, ACR, Investigational New Drug, Pharmaceutical industry, Vaccine, Nightclub, Cryptocurrency, Cabaletta, Limited

PHILADELPHIA, Nov. 09, 2023 (GLOBE NEWSWIRE) -- Cabaletta Bio, Inc. (Nasdaq: CABA), a clinical-stage biotechnology company focused on developing and launching the first curative targeted cell therapies for patients with autoimmune diseases, today reported financial results for the third quarter ended September 30, 2023, and provided a business update.

Key Points: 
  • In November 2023, Cabaletta announced the Company’s fourth IND application for CABA-201 was cleared by the FDA for a Phase 1/2 study in patients with generalized myasthenia gravis (gMG).
  • Cabaletta will present new preclinical data for CABA-201 in a poster presentation and Cabaletta Bio Scientific Advisory Board members Carl June, M.D., and Georg Schett, M.D.
  • Cabaletta plans to participate in the following upcoming investor conferences:
    Stifel 2023 Healthcare Conference, which is being held from November 14-15, 2023 in New York, NY.
  • As of September 30, 2023, Cabaletta had cash, cash equivalents and short-term investments of $164.4 million, compared to $106.5 million as of December 31, 2022.