SCID

Jasper Therapeutics Reports Fiscal 2022 Financial Results and Provides a Business Update

Retrieved on: 
Wednesday, March 8, 2023
Therapy, MDS, Sickle cell disease, Research, Conditional sentence, Fanconi anemia, Insurance, Growth, Hives, Public expenditure, Patient, R, Clinical trial, Research and development, Administration, CD117, SCID, Rare, Transplant, AML, B cell, Cash, Acute myeloid leukemia, Dentistry, Pharmaceutical industry, Cryptocurrency, Jasper

“By focusing on well-characterized opportunities in chronic mast cell diseases and stem cell transplant for rare diseases, we have established clear, and potentially fast, pathways to market.

Key Points: 
  • “By focusing on well-characterized opportunities in chronic mast cell diseases and stem cell transplant for rare diseases, we have established clear, and potentially fast, pathways to market.
  • Cash and Cash Equivalents: Cash and cash equivalents as of December 31, 2022 were $38.3 million.
  • The increase was primarily due to additional costs associated with advancing our clinical trials and clinical manufacturing expenses.
  • Net Loss: For the year ended December 31, 2022, net loss was $37.7 million compared to net loss of $30.6 million for the year ended December 31, 2021.

Jasper Therapeutics Announces Development Prioritization of Briquilimab in Chronic Diseases, Including Urticaria and Lower-Risk MDS, and Stem Cell Transplant for Sickle Cell Disease and Other Rare Diseases

Retrieved on: 
Tuesday, January 10, 2023
MDS, Toxicity, Therapy, Hives, Acute myeloid leukemia, Trial of the century, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Pharmacokinetics, B cell, Environment, Asthma, Skin, Conditional sentence, Lung, Fanconi anemia, Food, Degenerative disease, Patient, Transplant, Clinical trial, SCID, Sickle cell disease, Pharmaceutical industry, Jasper

This portfolio includes a new program on chronic urticaria, along with the Company’s existing programs for lower-risk myelodysplastic syndrome (MDS), sickle cell disease, Fanconi anemia and severe combined immunodeficiency (SCID).

Key Points: 
  • This portfolio includes a new program on chronic urticaria, along with the Company’s existing programs for lower-risk myelodysplastic syndrome (MDS), sickle cell disease, Fanconi anemia and severe combined immunodeficiency (SCID).
  • Based on preclinical and clinical studies showing inhibition of c-Kit signaling, depletion of mast cells in skin and lung and extended pharmacokinetics of subcutaneous dosing, the Company has prioritized rapidly starting a clinical study in severe chronic urticaria.
  • Clinical studies with briquilimab and investigational agents from other companies suggest that targeting c-Kit has strong therapeutic potential for chronic mast cell diseases such as urticaria and allergic asthma.
  • “We believe prioritizing these opportunities provides the best path forward to near-term, clinical milestones for patients and value creation for investors,” added Mr. Martell.

Jasper Therapeutics Announces European Union Orphan Drug Designation for Briquilimab as a Conditioning Treatment for Patients Prior to Receiving a Stem Cell Transplant

Retrieved on: 
Wednesday, January 4, 2023
Clinical trial, Bone marrow, Disorder, Food, Therapy, European Medicines Agency, HCT, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Blood, Haematopoietic system, Acute myeloid leukemia, CD117, Patient, Mortality, Committee, SCID, Prognosis, AML, Hematological Cancer Research Investment and Education Act, Microdeletion syndrome, Toxic epidermal necrolysis, Immune reconstitution inflammatory syndrome, EMA, MDS, COMP, Pharmaceutical industry, Vaccine, Jasper

Previously, the U.S. Food and Drug Administration granted orphan drug designation to briquilimab in HCT, as well as rare pediatric disease designation for the treatment of severe combined immunodeficiency (SCID).

Key Points: 
  • Previously, the U.S. Food and Drug Administration granted orphan drug designation to briquilimab in HCT, as well as rare pediatric disease designation for the treatment of severe combined immunodeficiency (SCID).
  • “The EMA’s decision to grant orphan drug designation to briquilimab highlights the clear need for non-genotoxic, targeted conditioning for patients receiving hematopoietic stem cell transplant,” said Ronald Martell, President and Chief Executive Officer of Jasper Therapeutics.
  • We believe that briquilimab has the potential to fill this gap, effectively expanding access to curative stem cell transplant across a range of indications.
  • Jasper’s ongoing clinical trial in SCID is evaluating briquilimab as a conditioning agent to enable stem cell transplantation in patients who are either transplant-naive or who received a prior stem cell transplant with a poor outcome.

PerkinElmer Announces its EONIS SCID-SMA Kit is First to Receive Marketing Authorization by U.S. FDA for SMA Screening in Newborns

Retrieved on: 
Monday, November 14, 2022
FDA, Other Health, Pharmaceutical, General Health, Medical Devices, Parenting, Children, Baby, Maternity, Family, Science, Biotechnology, Consumer, Other Science, Health, Research, Automation, FDA, Brainstem, Spinal muscular atrophy, Marketing, SCID, ESG, Food, Accreditation, Laboratory, Government, PerkinElmer, CE, PCR, DNA, Reproductive health, NYSE, Science, Motor neuron, SMA, Infant, Spinal cord, Software, Safety, Fundamental Analysis Software, Informatics, IVD, Birth, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, PKI, Degenerative disease, Inherited disorders of trafficking, Muscle weakness, Atrophy, Pharmaceutical industry

This is the first FDA authorized assay for SMA screening in newborns in the United States and is part of the Companys broader EONIS Platform .

Key Points: 
  • This is the first FDA authorized assay for SMA screening in newborns in the United States and is part of the Companys broader EONIS Platform .
  • This authorization is a major milestone for newborn screening in the United States.
  • Other components of the platform include the EONIS DNA Extraction kit and EONIS Analysis Software.
  • As the global leader in newborn screening, PerkinElmer offers solutions to help identify more than 50 congenital disorders.

Surrozen Presents Data on Two Lead Therapeutic Candidates at United European Gastroenterology (UEG) Week

Retrieved on: 
Monday, October 10, 2022
Institute of Liver and Biliary Sciences, Lung, NOAEL, Disease, U.S. Securities and Exchange Commission, Therapy, Liver disease, Degenerative disease, Edu, Annual report, Liver injury, Retina, SCID, Hepatocyte, RATS, Hepatitis, Research, Tissue, Regeneration, Security (finance), Cell proliferation, Eye, Soft tissue injury, SC, Inflammation, Nasdaq, Coronavirus, United European Gastroenterology, Mouse, Probability, Trial of the century, Human, Cornea, Alcoholic hepatitis, CEO, Liver, Skin, GLOBE, Ulcerative colitis, Fibrosis, Forward-looking statement, Inflammatory bowel disease, Homeostasis, No-observed-adverse-effect level, GLP, Toxicology, COVID-19, Pancreas, Dietary supplement, Pharmaceutical industry, WNT

Phase 1 clinical trials of our SZN-1326 and SZN-043 programs were initiated in May and June, respectively, and are ongoing.

Key Points: 
  • Phase 1 clinical trials of our SZN-1326 and SZN-043 programs were initiated in May and June, respectively, and are ongoing.
  • Surrozen believes that the transient, self-limited hepatocyte proliferation induced by SZN-043, is likely controlled by negative feedback loops responsible for regulation of cell proliferation and liver homeostasis.
  • SZN-043-related changes were considered non-adverse, limited to clinical chemistry and changes in organ weight and demonstrated evidence of recovery in most cases.
  • Surrozen is a clinical-stage biotechnology company discovering and developing drug candidates to selectively modulate the Wnt pathway.

Jasper Therapeutics Announces FDA Fast Track Designation for JSP191, a novel monoclonal antibody targeting CD117, in the treatment of patients with severe combined immunodeficiency (SCID) undergoing allogeneic hematopoietic stem cell transplant

Retrieved on: 
Thursday, September 15, 2022
Probability, Safety, Accelerated approval (FDA), COVID-19, Transplant, Risk, FA, SCID, FDA, Food, Stem cell, Biologics license application, RNA transfection, Acute myeloid leukemia, Population, Clinical trial, Review, Therapy, MDS, Forward-looking statement, Infection, Patient, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Anti-transglutaminase antibodies, Physician, Myelodysplastic syndrome, Biology, Sickle cell disease, Bone marrow, Fanconi anemia, Transfusion-dependent anemia, Toxic epidermal necrolysis, Annual report, Private Securities Litigation Reform Act, White Light Rock & Roll Review, Degenerative disease, Priority review, Fast Track, Collection, Microdeletion syndrome, AML, Lists of diseases, GLOBE, BLA, SEC, Immune system, Pharmaceutical industry, Vaccine, Jasper

JSP191 is a humanized monoclonal antibody that blocks stem cell factor receptor signaling leading to the clearance of hematopoietic stem and progenitor cells from the bone marrow.

Key Points: 
  • JSP191 is a humanized monoclonal antibody that blocks stem cell factor receptor signaling leading to the clearance of hematopoietic stem and progenitor cells from the bone marrow.
  • 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 hematopoietic cell transplantation.
  • While Jasper Therapeutics may elect to update these forward-looking statements at some point in the future, Jasper Therapeutics specifically disclaims any obligation to do so.

Asymmetrex's Introduction of Online Calculators for Determination of the Dosage of Therapeutic Stem Cells Announced as a Reformation in Stem Cell Science and Medicine

Retrieved on: 
Tuesday, September 13, 2022
Adult, Software, National Heart, Lung, and Blood Institute, SCID, KSC, LLC, Sherley, Mouse, Gold, Ministry of Education (Malaysia), Lung, CEO, Technology, Cell, B cell, Tissue, Drug development, Research, Medicine, R, Regenerative medicine, Wine, Fine chemical

BOSTON, Sept. 13, 2022 /PRNewswire-PRWeb/ -- In 2020, when Asymmetrex® published the seminal report of its innovation for counting therapeutic tissue stem cells for the first time, the company's President & CEO, James L. Sherley, M.D., Ph.D., says, "the method was already very good, but anything but routine."

Key Points: 
  • This month, stem cell biotechnology company Asymmetrex introduced the first online calculators for routine and rapid determination of the specific dosage of tissue stem cells not only in stem cell treatments, but also in stem cell biomanufacturing samples and stem cell research samples.
  • Asymmetrex has produced educational online forums and podcasts to reform ideas in stem cell science and stem cell medicine on the importance of attention to stem cell dosage for accelerating progress in stem cell research and therapy.
  • Says Sherley, "Our company's introduction of online rapid stem cell-counting calculators is the start of a reformation in stem cell science and medicine."
  • Asymmetrex markets kinetic stem cell (KSC) counting, the first technology for determination of the dose and quality of tissue stem cell preparations for use in stem cell transplantation medicine and pre-clinical drug evaluations.

ONK Therapeutics Presents Promising In-Vivo Data of its Optimized Affinity CD38 CAR-NK Candidate, Being Developed for the Treatment of Multiple Myeloma

Retrieved on: 
Monday, August 29, 2022
Oncology, Health, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, DR5, Cancer, CD96, NOD, NK, Tumor microenvironment, Survival, SCID, CAR, Therapy, Biotechnic & Histochemistry, Multiple myeloma, Mouse, Poster, CD38, CISH, NSG, CSO, TNF, Lymphatic system, JLab Audio, Cas9, CRISPR, DR4, Metabolism, Patient, Pharmaceutical industry, Medical imaging, Vaccine

ONKT102 is the companys most advanced program, and is being advanced towards clinical development as a potential treatment for patients with relapsed or refractory multiple myeloma (MM).

Key Points: 
  • ONKT102 is the companys most advanced program, and is being advanced towards clinical development as a potential treatment for patients with relapsed or refractory multiple myeloma (MM).
  • The anti-tumor efficacy of the optimized CD38 CAR-NK cells was then evaluated in NOD scid gamma (NSG) mice inoculated with MM.1S-LUC cells.
  • Prof. Michael ODwyer, founder and CSO at ONK Therapeutics said, These results suggest that non-virally engineered, optimized affinity CD38 CAR-NK CD38 KO cells have potent anti-tumor activity in-vitro and in-vivo in a CD38 positive tumor model.
  • ONK Therapeutics is headquartered in the med-tech hub of Galway, Ireland, with a wholly-owned US subsidiary, ONK Therapeutics, Inc. based at JLabs @ San Diego.

Jasper Therapeutics Reports Second Quarter 2022 Financial Results and Provides a Corporate Update

Retrieved on: 
Friday, August 12, 2022
MDS, CEO, Trial of the century, GLOBE, SEC, Clinical trial, Research, Annual report, Research and development, Biology, Toxic epidermal necrolysis, Transplant, Stem cell, TCT, Risk, Public expenditure, Stanford University School of Medicine, Safety, Anemia, BLA, Physician, RNA transfection, Bone marrow failure, FA, Forward-looking statement, Transfusion-dependent anemia, Growth, AML, Probability, Income, Holocene, Therapy, Bone marrow, COVID-19, Cash, Earnout, Degenerative disease, SCID, FDA, Acute myeloid leukemia, Definitive, Population, Microdeletion syndrome, Review, Company, Treatment, Lists of diseases, Private Securities Litigation Reform Act, Administration, Pharmaceutical industry, Vaccine, Jasper, Fanconi anemia, Patient

Research and Development (R&D) Expenses: R&D expenses for the quarter ended June 30, 2022 were $8.1 million compared to $5.2 million for the corresponding quarter in 2021.

Key Points: 
  • Research and Development (R&D) Expenses: R&D expenses for the quarter ended June 30, 2022 were $8.1 million compared to $5.2 million for the corresponding quarter in 2021.
  • If any of these risks materialize or Jasper Therapeutics assumptions prove incorrect, actual results could differ materially from the results implied by these forward-looking statements.
  • There may be additional risks that Jasper Therapeutics does not presently know, or that Jasper Therapeutics currently believes are immaterial, that could also cause actual results to differ from those contained in the forward-looking statements.
  • While Jasper Therapeutics may elect to update these forward-looking statements at some point in the future, Jasper Therapeutics specifically disclaims any obligation to do so.

Mustang Bio Announces First Patient Successfully Treated by Ex Vivo Lentiviral Gene Therapy to Treat RAG1 Severe Combined Immunodeficiency

Retrieved on: 
Wednesday, July 27, 2022
Patient, Immune reconstitution inflammatory syndrome, Haematopoietic system, GLOBE, Fever, U.S. Securities and Exchange Commission, Regulation, Orphan, Immunodeficiency, Forward-looking statement, SEC, CAR, Research, IND Culver Line, Partnership, Fortress Biotech, Securities Act of 1933, Private Securities Litigation Reform Act, Ownership, Securities Exchange Act of 1934, Lists of diseases, Failure, Patent, Immune system, SCID, NASDAQ, XSCID, Mustang, Pediatrics, Clinical trial, European Medicines Agency, Translation, RECOMB, Diarrhea, Rash, Annual report, Hematopoietic stem cell transplantation, Atlas Shrugged: Part I, Drug Quality and Security Act, RAG1, Infection, Leiden University Medical Center, Hematological Cancer Research Investment and Education Act, LUMC, Pharmaceutical industry, Vaccine

LV-RAG1 is exclusively licensed by Mustang for the development of MB-110, a first-in-class ex vivo lentiviral gene therapy for the treatment of RAG1-SCID.

Key Points: 
  • LV-RAG1 is exclusively licensed by Mustang for the development of MB-110, a first-in-class ex vivo lentiviral gene therapy for the treatment of RAG1-SCID.
  • The license agreement grants Mustang rights to certain additional lentiviral gene therapies being developed in Dr. Staals lab.
  • Severe combined immunodeficiency (SCID) due to complete RAG1 deficiency is a rare, genetic severe combined immunodeficiency disorder caused by null mutations in the RAG1 gene resulting in less than 1% of wild type V(D)J recombination activity.
  • Mustang has partnered with top medical institutions to advance the development of CAR T therapies across multiple cancers, as well as lentiviral gene therapies for severe combined immunodeficiency.