SCID

Jasper Therapeutics Announces Data from First Transplant-naive Patient in Phase 1 Clinical Trial of JSP191 as Conditioning Agent in Patients with SCID Undergoing Hematopoietic Cell Transplantation

Retrieved on: 
Monday, December 7, 2020
Oncology, Health, Stem cells, Other Health, Clinical trials, Pharmaceutical, Biotechnology, Medical specialties, Clinical medicine, Branches of biology, Stem cells, Transplantation medicine, Rare diseases, Hematology, Hematopoietic stem cell transplantation, Lymphology, Hematopoietic stem cell, Reticular dysgenesis, Severe combined immunodeficiency, SCID, JSP191, Jasper Therapeutics

The trial is evaluating JSP191 as a conditioning agent to enable stem cell transplantation in patients with SCID who are either transplant-naive or who received a prior stem cell transplant with a poor outcome.

Key Points: 
  • The trial is evaluating JSP191 as a conditioning agent to enable stem cell transplantation in patients with SCID who are either transplant-naive or who received a prior stem cell transplant with a poor outcome.
  • Hematopoietic cell transplantation offers the only curative therapy for SCID, a severe genetic immune disorder that leaves patients without a functioning immune system.
  • The open-label, multicenter Phase 1 study is evaluating the safety, tolerability and efficacy of JSP191 as a conditioning agent in patients with SCID undergoing first or repeat hematopoietic cell transplantation.
  • The companys lead compound, JSP191, is in clinical development as a conditioning antibody that clears hematopoietic stem cells from bone marrow in patients undergoing a hematopoietic cell transplant.

Mustang Bio and Minaris Regenerative Medicine Sign Technology Transfer and GMP Manufacturing Agreement for MB-107 Lentiviral Gene Therapy for X-Linked Severe Combined Immunodeficiency

Retrieved on: 
Monday, November 23, 2020
Medical specialties, Organ systems, Branches of biology, Emerging technologies, Rare diseases, Regenerative biomedicine, Regenerative medicine, Tissue engineering, Immunodeficiency, SCID, Severe combined immunodeficiency, X-linked severe combined immunodeficiency

This agreement with Minaris is an important step in supporting expansion of our MB-107 pivotal clinical trial into Europe, said Manuel Litchman, M.D., President and Chief Executive Officer of Mustang.

Key Points: 
  • This agreement with Minaris is an important step in supporting expansion of our MB-107 pivotal clinical trial into Europe, said Manuel Litchman, M.D., President and Chief Executive Officer of Mustang.
  • We look forward to working with Minaris to grow our geographic footprint and bring this potential life-saving therapy to XSCID patients in need internationally.
  • X-linked severe combined immunodeficiency is a rare genetic disorder that occurs in approximately 1 per 225,000 births.
  • Minaris Regenerative Medicine is a global contract development and manufacturing organization (CDMO) for cell and gene therapies.

Immune Deficiency Foundation launches website supporting parents of babies with SCID, a rare life-threatening disorder

Retrieved on: 
Tuesday, December 10, 2019
Rare diseases, Medical specialties, Medicine, Epidemiology, Neonatology, Newborn screening, Health, Severe combined immunodeficiency, SCID

IDF consulted with parents to build and review website content, employed the expertise of physicians in the writing and editing process, and utilized communication science teams to shape the website's core architecture.

Key Points: 
  • IDF consulted with parents to build and review website content, employed the expertise of physicians in the writing and editing process, and utilized communication science teams to shape the website's core architecture.
  • The launch of the website coincides with the one-year anniversary of all 50 states implementing screening for SCID in their newborn screening protocols.
  • That means a probable increase in the number of SCID cases and a greater need for information," said John G. Boyle, President and CEO of the Immune Deficiency Foundation.
  • The disorder is rare roughly 1 in 57,000 children are born with it annually, or about 76 cases in the U.S. each year.