Report of the 1st Annual Symposium on Relaxed Improvisation

Data From Incyte’s Povorcitinib Clinical Program to Be Featured at the 8th Annual Symposium on Hidradenitis Suppurativa Advances (SHSA)

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금요일, 10월 13, 2023
Health, Other Science, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, Abstract, Autoimmunity, Treatment, DLQI, Report of the 1st Annual Symposium on Relaxed Improvisation, Hidradenitis suppurativa, Drug development, Associate, Inflammation, HS, Pharmaceutical industry, Patient, Incyte

Incyte (Nasdaq:INCY) today announced that multiple abstracts featuring new data from the company’s clinical program on povorcitinib, an investigational oral JAK-1 inhibitor, have been accepted for oral presentation at the 8th Annual Symposium on Hidradenitis Suppurativa Advances (SHSA) held October 13-15 in Phoenix.

Key Points: 
  • Incyte (Nasdaq:INCY) today announced that multiple abstracts featuring new data from the company’s clinical program on povorcitinib, an investigational oral JAK-1 inhibitor, have been accepted for oral presentation at the 8th Annual Symposium on Hidradenitis Suppurativa Advances (SHSA) held October 13-15 in Phoenix.
  • ET)
    HiSQoL Changes Among HiSCR Responders and Nonresponders in a Phase 2 Study of Povorcitinib (Session: Clinical Research - Observational, Trials, and Treatment.
  • ET)
    Baseline Patient Characteristics Associated with Achieving HiSCR with Povorcitinib: Phase 2 Secondary Analysis (Session: Clinical Research - Observational, Trials, and Treatment.
  • ET)
    For full session details and data presentation listings, please see the SHSA 2023 ( https://shsa.joynsymposium.com/conference_hall ) online program.

Sonoma Biotherapeutics to Present Preclinical Data from Novel Treg Cell Therapy at Symposium on Hidradenitis Suppurativa Advances

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금요일, 10월 13, 2023
Health, Clinical Trials, Research, Pharmaceutical, Science, Biotechnology, Woman, Pain, Drainage, Beth Israel Deaconess Medical Center, Autoimmunity, Prevalence, Cell, MPH, Report of the 1st Annual Symposium on Relaxed Improvisation, Rheumatoid arthritis, CAR, Harvard Medical School, Protein, Black, Immune system, Scar, Hidradenitis suppurativa, Northwest Biotherapeutics, Skin, Inflammation, Patient, HS, Vaccine, Pharmaceutical industry, Regulatory T cell, Dermatology

Sonoma Biotherapeutics, Inc. , a clinical-stage biotechnology company developing engineered regulatory T cell (Treg) therapies for autoimmune and inflammatory diseases, will present preclinical data from its autologous chimeric antigen receptor (CAR) Treg product candidate for the treatment of hidradenitis suppurativa (HS) at the 8th Annual Symposium on Hidradenitis Suppurativa Advances (SHSA).

Key Points: 
  • Sonoma Biotherapeutics, Inc. , a clinical-stage biotechnology company developing engineered regulatory T cell (Treg) therapies for autoimmune and inflammatory diseases, will present preclinical data from its autologous chimeric antigen receptor (CAR) Treg product candidate for the treatment of hidradenitis suppurativa (HS) at the 8th Annual Symposium on Hidradenitis Suppurativa Advances (SHSA).
  • “CAR-Treg therapies hold great promise for treating an array of autoimmune and inflammatory diseases,” said Mark D. Eisner, M.D., MPH, Chief Medical Officer of Sonoma Biotherapeutics.
  • “Our 7101 program targets citrullinated proteins, a hallmark of many autoimmune diseases, including rheumatoid arthritis and HS.
  • Our results show that we can engineer CAR Tregs directly from HS patients’ cells that can target these proteins.

UCB Reinforces Commitment to Advancing Care in Hidradenitis Suppurativa with Six Abstracts at SHSA 2023

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금요일, 10월 13, 2023
Diagnosis, Food, Abstract, Research, UCB, Report of the 1st Annual Symposium on Relaxed Improvisation, Insurance, Safety, Bimekizumab, Hidradenitis suppurativa, Policy, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, BE, Pharmaceutical industry, Pain, Patient, Political moderate, HS

ATLANTA, Oct. 13, 2023 /PRNewswire/ -- UCB, a global biopharmaceutical company, today announced that it will present six abstracts in hidradenitis suppurativa (HS) at the 8th Annual Symposium on Hidradenitis Suppurativa Advances (SHSA), October 13–15 in Phoenix, Arizona.

Key Points: 
  • ATLANTA, Oct. 13, 2023 /PRNewswire/ -- UCB, a global biopharmaceutical company, today announced that it will present six abstracts in hidradenitis suppurativa (HS) at the 8th Annual Symposium on Hidradenitis Suppurativa Advances (SHSA), October 13–15 in Phoenix, Arizona.
  • "The data to be presented at SHSA reinforce our commitment to advancing medicines in areas where patients have the greatest need," said Emmanuel Caeymaex, Executive Vice President, Immunology Solutions and Head of U.S., UCB.
  • In a second oral presentation, 48-week data evaluating the impact of bimekizumab on health-related quality of life will be shared.
  • Three poster presentations will include subgroup analyses from the two Phase 3 studies, including 48-week data in U.S. patients.

FDA awards nearly $7.5 million to pediatric device consortium led by Children’s National Hospital

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화요일, 9월 19, 2023
Walter Reed Army Medical Center, Therapy, Report of the 1st Annual Symposium on Relaxed Improvisation, Elective surgery, University, FDA Center for Devices and Radiological Health, Research, African Americans, Food and Drug Administration Amendments Act of 2007, Acquisition, PDC, Tufts Medical Center, Johns Hopkins University, Mass, Mass General Brigham, AdvaMed, RWE, World Creativity and Innovation Day, Ecosystem, FDA, Health, Device, Conference, Food, Harwell Science and Innovation Campus, Birth control, Pharmaceutical industry

WASHINGTON, D.C., Sept. 19, 2023 (GLOBE NEWSWIRE) -- To foster the development and commercialization of medical devices designed for children, the Food and Drug Administration (FDA) has awarded a nearly $7.5 million grant to the Alliance for Pediatric Device Innovation (APDI), a consortium led by Children’s National Hospital .

Key Points: 
  • WASHINGTON, D.C., Sept. 19, 2023 (GLOBE NEWSWIRE) -- To foster the development and commercialization of medical devices designed for children, the Food and Drug Administration (FDA) has awarded a nearly $7.5 million grant to the Alliance for Pediatric Device Innovation (APDI), a consortium led by Children’s National Hospital .
  • New in this cycle, APDI will provide expertise on evidence generation, including the use of real-world evidence (RWE), for pediatric device development.
  • Along with Children’s National, APDI consortium members include Johns Hopkins University, CIMIT at Mass General Brigham, Tufts Medical Center and Medstar Health Research Institute.
  • Eskandanian says the new Children’s National Research and Innovation Campus provides the ideal environment for the collaborative work needed to advance pediatric innovation.

Orchard Therapeutics Announces Acceptance of Biologics License Application for OTL-200 in MLD and Receives Priority Review

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월요일, 9월 18, 2023
Priority review, Society, Natural history, Orchard, MLD, San Raffaele Hospital, Death, Prescription Drug User Fee Act, Patient, Data, Child, Busulfan, FDA, Therapy, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, BLA, Report of the 1st Annual Symposium on Relaxed Improvisation, DSM-IV codes, Food, Disease, Pharmaceutical industry, Metabolism, PDUFA

BOSTON and LONDON, Sept. 18, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the U.S. Food and Drug Administration (FDA) has accepted the filing of its Biologics License Application (BLA) for OTL-200 in metachromatic leukodystrophy (MLD) under Priority Review. The agency has set a Prescription Drug User Fee Act (PDUFA) goal date of March 18, 2024.

Key Points: 
  • The agency has set a Prescription Drug User Fee Act (PDUFA) goal date of March 18, 2024.
  • “We look forward to collaborating with the FDA throughout the review and evaluation of our application.
  • With more than a cumulative 250 patient-years of follow-up, treatment with OTL-200 was generally well-tolerated, with no treatment-related serious adverse events or deaths.
  • The full clinical results comprising the BLA dataset were recently presented at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium 2023 in Jerusalem.

Acepodia Announces Nobel Laureate Carolyn Bertozzi, Ph.D., as Chief Scientific Advisor

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월요일, 9월 18, 2023
ACC, Nobel Prize, National academy, Stanford University, Nobel Prize in Chemistry, Cancer, Coronavirus Scientific Advisory Board, Howard Hughes Medical Institute, Cell, Click chemistry, Hematological Cancer Research Investment and Education Act, Patient, Nobel, Anne, Report of the 1st Annual Symposium on Relaxed Improvisation, Pharmaceutical industry, Medical device, Chemistry, Invention

ALAMEDA, Calif. and TAIPEI, Sept. 18, 2023 /PRNewswire/ -- Acepodia, a clinical stage biotechnology company developing first-in-class cell therapies with its unique Antibody-Cell Conjugation (ACC) and allogeneic gamma delta 2 T-cell platforms to address gaps in cancer care, today announced that Nobel Laureate Carolyn Bertozzi, Ph.D., was recently appointed Chief Scientific Advisor.

Key Points: 
  • ALAMEDA, Calif. and TAIPEI, Sept. 18, 2023 /PRNewswire/ -- Acepodia, a clinical stage biotechnology company developing first-in-class cell therapies with its unique Antibody-Cell Conjugation (ACC) and allogeneic gamma delta 2 T-cell platforms to address gaps in cancer care, today announced that Nobel Laureate Carolyn Bertozzi, Ph.D., was recently appointed Chief Scientific Advisor.
  • Dr. Bertozzi's appointment was celebrated at Acepodia's Annual Symposium in San Francisco Bay Area, CA on September 13.
  • Dr. Bertozzi was awarded the 2022 Nobel Prize in Chemistry for discovering a novel form of click chemistry, known as bioorthogonal chemistry.
  • As Chief Scientific Advisor, Dr. Bertozzi will lead Acepodia's Scientific Advisory Board in guiding the company's scientific progress and offering insights based on their relevant experience.

Denali Therapeutics Announces New Interim Data from Phase 1/2 Study of DNL310 (ETV:IDS) in MPS II (Hunter Syndrome) at SSIEM 2023

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수요일, 8월 30, 2023
Cognition, Learning, Partnership, Society, Event, AFL, Serum, Genomics, ABR, Glycogen storage disease, Hunting, MPS II, Traffic barrier, COMPASS, Biomarker, MPS, Hunter syndrome, BBB, Anne, Patient, Hearing, CSF, ETV, PDF, Infant, Nerve, Report of the 1st Annual Symposium on Relaxed Improvisation, Safety, Disease, Medical imaging, Pharmaceutical industry, Dietary supplement, Metabolism, Genetics, MD

DNL310 is an investigational enzyme replacement therapy designed to cross the BBB and address the behavioral, cognitive, and physical manifestations of MPS II.

Key Points: 
  • DNL310 is an investigational enzyme replacement therapy designed to cross the BBB and address the behavioral, cognitive, and physical manifestations of MPS II.
  • The interim data from the Phase 1/2 study of DNL310 were highlighted in an oral presentation at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium 2023 in Jerusalem, Israel.
  • A PDF of the Phase 1/2 presentation is available on Denali’s website on the Events page of the Investor section.
  • We are encouraged to see positive changes across multiple clinical outcomes measures in the ongoing Phase 1/2 study.

Data from Glycomine’s Ongoing Natural History Study Provides Insight into Clinical Measures and Biomarkers for PMM2-CDG

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수요일, 8월 30, 2023
Health, Clinical Trials, Research, Pharmaceutical, Science, Biotechnology, Society, Mayo Clinic, Biomarker, Patient, Liver, PMM2-CDG, Principal, Report of the 1st Annual Symposium on Relaxed Improvisation, Caregiver, Disease, Pharmaceutical industry, Metabolism

The findings included baseline values of key coagulation, liver, metabolic, and endocrine measurements and their evolution over two to four years.

Key Points: 
  • The findings included baseline values of key coagulation, liver, metabolic, and endocrine measurements and their evolution over two to four years.
  • “We are grateful for the time and commitment contributed by the patients and their caregivers and appreciative of the investigators and clinical sites conducting the study,” said Steven Axon, CEO of Glycomine.
  • Every patient currently enrolled has completed more than two years in the study.
  • The effort has resulted in the largest single dataset in patients with PMM2-CDG from a prospective natural history study to date.

Initial Clinical Data of First Pediatric CLN2 Patient Dosed with RGX-181 Presented at SSIEM Annual Symposium

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수요일, 8월 30, 2023
Seizure, Society, Therapy, TPP1, Neurodegenerative disease, Kia Challenge, Gene expression, Accelerated approval (FDA), Language, AAV, Hunter syndrome, Batten disease, Death, ERT, Patient, Child, Hospital, Enzyme replacement therapy, Safety, CLN2, BLA, Report of the 1st Annual Symposium on Relaxed Improvisation, Peptide, Disease, Pharmaceutical industry, Metabolism

"CLN2 is a debilitating disease caused by mutations in the CLN2 gene resulting in a deficiency of the TPP1 enzyme, which is needed to break down specific peptides associated with cellular waste.

Key Points: 
  • "CLN2 is a debilitating disease caused by mutations in the CLN2 gene resulting in a deficiency of the TPP1 enzyme, which is needed to break down specific peptides associated with cellular waste.
  • "We are encouraged by the initial results demonstrating that RGX-181 is well tolerated and dramatically reduced the number of seizures in the patient enrolled in this trial."
  • "The remarkable decrease in seizures, encouraging safety results and reduction in ERT frequency highlight the potential of this gene therapy to provide a meaningful treatment option to the CLN2 patient community."
  • Today, a physician investigator from the Hospital de Clinicas in Porto Alegre, Brazil reported initial results from a five-year-old child who received a one-time intracisternal dose of RGX-181.

Travere Therapeutics to Present Abstracts at the Society for the Study of Inborn Errors of Metabolism Annual Symposium 2023

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목요일, 8월 24, 2023
DSM-IV codes, Society, Prevalence, Homocystinuria, Burden, ICC, HCU, IDT, Exhibition Hall of the Ministry of Culture, Cystathionine beta synthase, SAN, Therapy, Report of the 1st Annual Symposium on Relaxed Improvisation, Pharmaceutical industry, Metabolism

SAN DIEGO, Aug. 24, 2023 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc. (NASDAQ: TVTX) today announced that the Company will present clinical data from the Phase 1/2 COMPOSE Study of pegtibatinase, a novel investigational enzyme replacement therapy being evaluated for the treatment of classical homocystinuria (HCU), at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium in Jerusalem, Israel, August 29 – September 1, 2023.

Key Points: 
  • SAN DIEGO, Aug. 24, 2023 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc. (NASDAQ: TVTX) today announced that the Company will present clinical data from the Phase 1/2 COMPOSE Study of pegtibatinase, a novel investigational enzyme replacement therapy being evaluated for the treatment of classical homocystinuria (HCU), at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium in Jerusalem, Israel, August 29 – September 1, 2023.
  • The Company and its collaborators will also present analyses on its prospective HCU natural history study, the prevalence of HCU, and the burden of HCU from the patient perspective highlighting the devastating nature of this rare metabolic disease over patients’ lifetimes and underscoring the urgent need for new treatments.
  • In addition, the Company will present data on the clinical burden of HCU and the relationship between total homocysteine (tHcy) and clinical outcomes, which has been recognized as one of the highest ranked posters at SSIEM.
  • Clinical Characterization of Classical Homocystinuria Due to Cystathionine-beta Synthase Deficiency: Results from the ACAPPELLA Study
    Clinical Burden of Classical Homocystinuria in the United States: A Retrospective Analysis of Optum Market Clarity
    Understanding the Burden of Classical Homocystinuria (HCU) from the Patient’s Perspective: A Qualitative Study