SCID

Smart Immune Announces Research Collaboration with Memorial Sloan Kettering Cancer Center (MSK) to Expedite Development of a Next Generation CAR-T Through its Long Lasting, Exhaustion-Free CAR ProTcell™ Platform

Retrieved on: 
Thursday, December 9, 2021
HSC, Research, Neoplasm, GLOBE, Technology, Immune reconstitution inflammatory syndrome, Mortality, Culture, Investigational New Drug, Moirangthem Maniram Singha, POC, Immune system, IND, Mouse, Patient, AML, Disease, ALL, Division, MSK, Graft-versus-host disease, NSG, Lymphatic system, CD19, Cancer, Memorial Sloan Kettering Cancer Center, Program, Acute leukemia, SCID, FDA, HSCT, Immunodeficiency, Prognosis, Infection, Transplant, Vaccine

This collaboration has been setup to rapidly develop the next generation of CAR-T cell therapies.

Key Points: 
  • This collaboration has been setup to rapidly develop the next generation of CAR-T cell therapies.
  • Through its groundbreaking ex vivo thymus technology, Smart Immune aims to disrupt conventional approaches to CAR-T cell therapies by using its T-cell progenitors ie.
  • ProTcell instead of mature T-cell and generate a nave long lasting, exhaustion-free CAR-T cell population.
  • ProTcell is currently being studies in two clinical trials in Europe, with two in the U.S. expected to start in early 2022.

Jasper Therapeutics to Present Data on JSP191 Conditioning in SCID patients at the 2021 American Society of Hematology Annual Meeting

Retrieved on: 
Wednesday, December 8, 2021
HSC, Physician, Probability, Lists of diseases, Risk, Forward-looking statement, Research and development, Population, SEC, GLOBE, Severe combined immunodeficiency, Immune reconstitution inflammatory syndrome, Failure, Safety, Private Securities Litigation Reform Act, Degenerative disease, Research, RNA transfection, Therapy, Bone marrow, Â, Business architecture, Transplant, COVID-19, SCID, HCT, NASDAQ, Lymphocyte, Stem cell, Half-life, Biology, Annual report, Society, ASH, Vaccine, Pharmaceutical industry

Title: JSP191 As a Single-Agent Conditioning Regimen Results in Successful Engraftment, Donor Myeloid Chimerism, and Production of Donor Derived Nave Lymphocytes in Patients with Severe Combined Immunodeficiency (SCID)

Key Points: 
  • Title: JSP191 As a Single-Agent Conditioning Regimen Results in Successful Engraftment, Donor Myeloid Chimerism, and Production of Donor Derived Nave Lymphocytes in Patients with Severe Combined Immunodeficiency (SCID)
    Session: 721.
  • Jasper Therapeutics is a biotechnology company focused on the development of novel curative therapies based on the biology of the hematopoietic stem cell.
  • AMHC and Jasper Therapeutics anticipate that subsequent events and developments will cause AMHCs and Jasper Therapeutics assessments to change.
  • These forward-looking statements should not be relied upon as representing AMHCs and Jasper Therapeutics assessments of any date subsequent to the date of this press release.

Jasper Therapeutics Announces Third Quarter 2021 Financial Results and Provides Business Update

Retrieved on: 
Friday, November 12, 2021
Stem cell, Safety, AML, Bone marrow, MRNA surveillance, Private Securities Litigation Reform Act, Therapy, Lists of diseases, RNA transfection, Microdeletion syndrome, SCID, Clinical trial, Engineering, Toxic shock syndrome, Prospectus, Security (finance), Cash, NCI, Autoimmune disease, Growth, MDS, Fabry disease, Administration, COVID-19, Research, Securities Act of 1933, Forward-looking statement, Public expenditure, Transplant, Research and development, National Cancer Institute, Company, U.S. Securities and Exchange Commission, GLOBE, Degenerative disease, SEC, Drug Quality and Security Act, Biology, Nasdaq, Patient, Risk, National, Pharmaceutical industry, Vaccine

REDWOOD CITY, Calif., Nov. 12, 2021 (GLOBE NEWSWIRE) -- Jasper Therapeutics, Inc. (Nasdaq: JSPR), a biotechnology company focused on hematopoietic cell transplant therapies, today announced results for the quarter ended September 30, 2021, and provided a business update.

Key Points: 
  • REDWOOD CITY, Calif., Nov. 12, 2021 (GLOBE NEWSWIRE) -- Jasper Therapeutics, Inc. (Nasdaq: JSPR), a biotechnology company focused on hematopoietic cell transplant therapies, today announced results for the quarter ended September 30, 2021, and provided a business update.
  • Cash and Cash Equivalents: Cash and cash equivalents as of September 30, 2021, were $100.9 million.
  • The Company expects current cash and cash equivalents to be sufficient to fund its planned operating and capital expenditures through mid-2023.
  • Jasper Therapeutics is a biotechnology company focused on the development of novel curative therapies based on the biology of the hematopoietic stem cell.

Mustang Bio Announces Exclusive Worldwide License Agreement with Leiden University Medical Centre for Clinical-Stage Lentiviral Gene Therapy with Curative Potential for RAG1 Severe Combined Immunodeficiency

Retrieved on: 
Wednesday, November 10, 2021
Health, Securities Exchange Act of 1934, Leiden University Medical Center, Private Securities Litigation Reform Act, XSCID, Fortress Biotech, Lists of diseases, Immunodeficiency, Hematopoietic stem cell transplantation, SCID, Clinical trial, U.S. Securities and Exchange Commission, Education, NASDAQ, Ownership, Failure, HSCT, Immune reconstitution inflammatory syndrome, Rash, European Medicines Agency, Infection, LUMC, Research, CAR, Securities Act of 1933, Forward-looking statement, Regulation, Partnership, Patient, Mustang, Translation, GLOBE, Hematological Cancer Research Investment and Education Act, SEC, Fever, Diarrhea, Lymphocytopenia, Patent, RAG1, Pharmaceutical industry

WORCESTER, Mass., Nov. 10, 2021 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. (“Mustang”) (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, today announced that the company has executed an exclusive license agreement with Leiden University Medical Centre (“LUMC”) for a first-in-class ex vivo lentiviral gene therapy for the treatment of RAG1 severe combined immunodeficiency (“RAG1-SCID”).

Key Points: 
  • Dr. Staal will continue the development of additional lentiviral gene therapies in his lab, to which Mustang Bio has rights under the agreement.
  • The RAG1-SCID therapy expands the pipeline of ex vivo lentiviral gene therapies currently in development at Mustang.
  • The Companys lead programs, MB-107 and MB-207, are being investigated for the treatment of X-linked severe combined immunodeficiency (XSCID).
  • Mustang is establishing itself as the leader in developing treatments for patients with severe combined immunodeficiency, an area of high unmet need.

Jasper Therapeutics Announces Research Collaboration with AVROBIO to Evaluate JSP191 as Conditioning Agent in Clinical Studies of Ex Vivo Lentiviral Gene Therapy

Retrieved on: 
Tuesday, November 9, 2021
Stem cell, Safety, AML, Bone marrow, Private Securities Litigation Reform Act, Therapy, Gaucher, Lists of diseases, Microdeletion syndrome, SCID, Prospectus, Lymphatic system, Security (finance), Autoimmune disease, MDS, Fabry disease, COVID-19, Fanconi anemia, Securities Act of 1933, Forward-looking statement, Jasper, Transplant, U.S. Securities and Exchange Commission, GLOBE, Sickle cell disease, Degenerative disease, SEC, Chronic granulomatous disease, Drug Quality and Security Act, GATA2, Biology, Patient, Risk, Pharmaceutical industry, Vaccine

This collaboration with AVROBIO further expands the potential of JSP191 for use with lentiviral gene therapies, said Bill Lis, executive chairman and chief executive officer of Jasper Therapeutics.

Key Points: 
  • This collaboration with AVROBIO further expands the potential of JSP191 for use with lentiviral gene therapies, said Bill Lis, executive chairman and chief executive officer of Jasper Therapeutics.
  • Jasper is focused on building a leading hematopoietic stem cell therapeutics company with the potential to bring one-time treatments to patients with blood cancers, rare monogenic diseases and autoimmune diseases.
  • Jasper Therapeutics is a biotechnology company focused on the development of novel curative therapies based on the biology of the hematopoietic stem cell.
  • JSP191, an anti-CD117 monoclonal antibody, is in clinical development as a conditioning agent that clears hematopoietic stem cells from bone marrow in patients undergoing a hematopoietic cell transplantation.

Cellectis Presents Initial Preclinical Data on Two Novel Gene Therapies for Patients with RAG1 Severe Combined Immunodeficiency (SCID) and Hyper IgE syndrome at ESGCT 2021

Retrieved on: 
Tuesday, October 19, 2021
European Society of Gene and Cell Therapy, STAT3, RAG1, AML, GLOBE, Patient, University, Glycogen storage disease, ESGCT, Immunodeficiency, Lists of diseases, SCID, Gene, TALEN, Risk, Private Securities Litigation Reform Act, COVID-19, LinkedIn, Skin, Acute lymphoblastic leukemia, YouTube, Population, Sickle cell disease, CAR, Urinary tract infection, Stem cell, Health, Center, Technology, Degenerative disease, World Creativity and Innovation Day, Gene editing, Social media, Annual report, Disease, U.S. Securities and Exchange Commission, B-ALL, Securities & Exchange Commission of Pakistan, Multiple myeloma, Forward-looking statement, University of Freiburg, Cancer, Congress, NASDAQ, Euronext Growth, Conradin Cathomen, Cell, HSC, Research, Acute myeloid leukemia, Nasdaq, Vaccine, Antibiotics

ProfessorCathomensteam at University of Freiburgwill bepresentingencouraging pre-clinical data that supports further evaluation ofCellectis .HEAL platform,aninnovative gene therapyplatformthat usesa genome editing approachbased onTALEN ,fortwoproduct candidatestargetingprimary immunodeficiencies: RAG1forSevere Combined Immunodeficiency (SCID) andSTAT3 forHyperIgEsyndrome.

Key Points: 
  • ProfessorCathomensteam at University of Freiburgwill bepresentingencouraging pre-clinical data that supports further evaluation ofCellectis .HEAL platform,aninnovative gene therapyplatformthat usesa genome editing approachbased onTALEN ,fortwoproduct candidatestargetingprimary immunodeficiencies: RAG1forSevere Combined Immunodeficiency (SCID) andSTAT3 forHyperIgEsyndrome.
  • Last May,duringCellectis Innovation Days ,the Companyrevealed its new .HEAL platform, a novel hematopoietic stem cell gene therapy that aims to address debilitating genetic diseases.
  • Newborns with RAG1 SCID have extremely low levels of B and T cells and a severe risk of recurrent, life-threatening infections.
  • The precise replacement of the mutated gene enabled the corrected RAG1 gene to be expressed at its natural timing and stage of cell development.

FDA Approves Innovative Treatment for Pediatric Patients with Congenital Athymia

Retrieved on: 
Friday, October 8, 2021
Center for Biologics Evaluation and Research, MedWatch, Public health, Risk, Therapy, Office of Inspector General, U.S. Department of Health and Human Services, Life, Cytokine release syndrome, Food, Multimedia, Survival, Safety, Guidance, Research, Human services, FDA, Child, Birth defect, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Immune reconstitution inflammatory syndrome, Infection, Body surface area, Dietary supplement, Immune disorder, Rash, Vaccine, Health care, Smokeless tobacco, Animal, Cosmetics, Patient, Disease, Immunity, China Biologic Products, Center, SCID, Pharmaceutical industry, Generic drug

SILVER SPRING, Md., Oct. 8, 2021 /PRNewswire/ -- Today, the U.S. Food and Drug Administration approved Rethymic for the treatment of pediatric patients with congenital athymia, a rare immune disorder.

Key Points: 
  • SILVER SPRING, Md., Oct. 8, 2021 /PRNewswire/ -- Today, the U.S. Food and Drug Administration approved Rethymic for the treatment of pediatric patients with congenital athymia, a rare immune disorder.
  • Congenital athymia is a rare immune disorder in which a child is born without a thymus an organ that plays a critical role in helping the body learn to fight infections.
  • Rethymic improved survival of children with congenital athymia, and most children treated with this product survived at least two years.
  • A description of rare pediatric disease designation and voucher programs can be found in the Guidance for Industry: Rare Pediatric Disease Priority Review Vouchers .

Aviceda Therapeutics Announces Additional World-Class Members to its Scientific Advisory Board

Retrieved on: 
Monday, September 20, 2021
Technology, Research, Infectious Diseases, Nanotechnology, Clinical Trials, Biotechnology, Health, Pharmaceutical, Science, Therapy, VU University Medical Center, JAK3, List of pathologists, KNAW, Dendrite, ERC, Cell, Royal Netherlands Academy of Arts and Sciences, Harvard Medical School, SCID, Aviceda, Inflammation, Pathology, Coronavirus Scientific Advisory Board (Turkey), Cell biology, IP, Immune system, Cancer research, Science, Amgen, JEM, HIV, COO, GA, UCLA, FIH, CSO, Molecular biology, Ghost, DNA repair, DNA, Geographic atrophy, Government, Autosome, Cancer Research UK, Doctor of Philosophy, College, Government of Northern Ireland, Society, Dendritic cell, College of American Pathologists, DNAR, II, NWO, Cancer, CLIA, Patient, Biomarker, Biology, CLEP, McClay, NIH, PNAS, Pharmaceutical industry, Vaccine, Medicine

Aviceda Therapeutics, an IND-stage, pre-clinical biotech company focused on developing the next generation of immuno-modulators by harnessing the power of glycobiology to manipulate the innate immune system and chronic, non-resolving inflammation, is announcing additional members of its Scientific Advisory Board who will help shape ongoing research and development efforts.

Key Points: 
  • Aviceda Therapeutics, an IND-stage, pre-clinical biotech company focused on developing the next generation of immuno-modulators by harnessing the power of glycobiology to manipulate the innate immune system and chronic, non-resolving inflammation, is announcing additional members of its Scientific Advisory Board who will help shape ongoing research and development efforts.
  • The new additional Aviceda Scientific Advisory Board members includes:
    Aviceda is honored and privileged to add new world-class scientists and renowned researchers to join our efforts in developing the next generation of glyco-immune therapeutics for the treatment of immune-dysfunction conditions, said Mohamed A. Genead, MD, Co-Founder & President of Aviceda Therapeutics.
  • She published more than 280 scientific papers in well recognized journals (Cell, Nature, Nature Immunology, PNAS, and JEM) and supervised 29 PhD students.
  • The Aviceda team is already building on the foundational work in the emerging field of glycobiology to develop potential therapeutics and interventional strategies.

Jasper Therapeutics Initiates New Clinical Trial with National Cancer Institute to Evaluate JSP191 in GATA2-related Myelodysplastic Syndromes

Retrieved on: 
Wednesday, September 8, 2021
Research, Clinical Trials, Biotechnology, Health, Pharmaceutical, General Health, Other Science, Science, Oncology, JSP191, Jasper Therapeutics, JSP191, JASPER THERAPEUTICS

Jasper Therapeutics and the National Cancer Institute (NCI), part of the National Institutes of Health, have entered into a clinical trial agreement in which NCI will serve as the Investigational New Drug (IND) sponsor for this study.

Key Points: 
  • Jasper Therapeutics and the National Cancer Institute (NCI), part of the National Institutes of Health, have entered into a clinical trial agreement in which NCI will serve as the Investigational New Drug (IND) sponsor for this study.
  • The results may provide us with key insights about the use of JSP191, a highly differentiated anti-CD117 monoclonal antibody, as a conditioning agent for this patient population.
  • Two clinical trials for myelodysplastic syndromes (MDS)/acute myeloid leukemia (AML) and severe combined immunodeficiency (SCID) are currently enrolling.
  • Jasper Therapeutics is a biotechnology company focused on the development of novel curative therapies based on the biology of the hematopoietic stem cell.

Enzyvant Announces First-Ever Data on Burden of Illness and Costs of Supportive Care for Pediatric Congenital Athymia

Retrieved on: 
Thursday, August 26, 2021
ATMP, EMA, SCID, Life, Immunodeficiency, Family, Trauma, Caregiver, Food, T cell deficiency, Journal of Medical Economics, Immunology, Health care, Orphan drug, Immunosuppression, Time, Child, Sibling, European Medicines Agency, Sumitomo Dainippon Pharma, Hospital, Patient, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Friends, Twitter, Incidence, Diagnosis, Pediatrics, Advanced Therapy Medicinal Product, Sport, LinkedIn, FDA, Ministry of Education (Malaysia), Advances in Therapy, Journal, HCPS, Health, Birth defect, Literature, GLOBE, Infection, Physician, PDUFA, Fear, NYSE, BLA, Prescription Drug User Fee Act, Infant, Parent, Residential care, Pharmaceutical industry, Medicine, Dentistry, Health insurance, Machine tool

CAMBRIDGE, Mass. and BASEL, Switzerland, Aug. 26, 2021 (GLOBE NEWSWIRE) -- Data from two first-ever studies reveal extreme clinical, emotional, social, and financial burdens on patients with pediatric congenital athymia and their families, and extraordinary costs of supportive care to healthcare systems. Congenital athymia is an ultra-rare condition in which children are born without a thymus, leading to profound immunodeficiency, life-threatening immune dysregulation, and high susceptibility to potentially fatal infections. Estimated incidence of congenital athymia in the U.S. is ~17 to 24 live births each year.1 Currently, there are no FDA-approved treatments for congenital athymia.

Key Points: 
  • Supportive care is used to prevent potentially fatal infections and manage other symptoms of immunodeficiency and immune dysregulation due to congenital athymia.
  • With only supportive care, patients with congenital athymia typically die by age two or three.
  • To evaluate the burdens of congenital athymia on patients and families, and the total medical costs associated with supportive care, two separate studies were conducted.
  • Both the congenital athymia caregiver-assessed burden of illness and cost of supportive care studies were fully supported with funding from Enzyvant.