Severe Combined Immunodeficiency News and Research

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Scientists launch clinical trial of CRISPR gene correction therapy in patients with sickle cell disease

Scientists at UC San Francisco, UC Berkeley and UCLA have received U.S. Food and Drug Administration approval to jointly launch an early phase, first-in-human clinical trial of a CRISPR gene correction therapy in patients with sickle cell disease using the patient's own blood-forming stem cells.

30 Mar 2021

New test will help better manage patients with severe combined immunodeficiency

A new test developed at CHU Sainte-Justine in Montreal will enable better management of patients with severe combined immunodeficiency.

17 Jun 2020

Research team generates comprehensive developmental map of human T-cells

Chintan Parekh, MD, of The Saban Research Institute of Children's Hospital Los Angeles, has led a team of investigators that generated a comprehensive roadmap for how T-cells develop in the human thymus.

16 Jun 2020

COVID-19 and immunodeficiency

COVID-19 disease, caused by severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), is a highly infectious disease. As of today, over 170,000 people worldwide have succumbed to the infection. Reports have shown that some comorbidities and illnesses raise the risk of getting infected and also influence the severity of the disease. Having a compromised immune system can also drastically raise the risk of getting COVID-19.

21 Apr 2020

NIH grants boost rare diseases research efforts

Of an estimated 6,500 to 7,000 known rare diseases, only a fraction - maybe 5% - have U.S. Food and Drug Administration-approved treatments.

4 Oct 2019

Despite failed promises, stem cell advocates again want taxpayers to pony up billions

Californians voted in 2004 to shell out billions of dollars in taxpayer money to fund cutting-edge stem cell treatments.

16 Aug 2019

New gene therapy cures babies with fatal 'Bubble Boy' disease

A new gene therapy created by US researchers appears to have cured eight infant boys born with a rare genetic disorder referred to as “Bubble Boy” disease.

18 Apr 2019

Antibody-based treatment could improve cure rate for blood and immune disorders

An antibody-based treatment can gently and effectively eliminate diseased blood-forming stem cells in the bone marrow to prepare for the transplantation of healthy stem cells, according to a study in mice by researchers at the Stanford University School of Medicine.

12 Feb 2019

Leadiant Biosciences announces FDA approval of Revcovi for treating pediatric, adult ADA-SCID patients

Leadiant Biosciences, Inc. today announced that the Food and Drug Administration has granted approval to Revcovi (elapegademase-lvlr) injection in the U.S. Revcovi is a new enzyme replacement therapy for the treatment of adenosine deaminase severe combined immune deficiency in pediatric and adult patients.

7 Oct 2018

Using novel gene therapy to cure infants born with severe combined immunodeficiency

Out of every 60,000 births, a baby arrives to face the world without a fully functioning immune system leaving them unequipped to fight even the most common infections.

29 Aug 2018

Smart Cells launches social campaign to support Rare Disease Day 2018

There are more than 6000 known rare diseases and over 30 million people living daily with them. 28th February marks Rare Disease Day 2018, a date that highlights awareness around these conditions and the need for more research into them.

From Smart Cells International Ltd 23 Feb 2018

A guide to Severe Combined Immunodeficiency Disease, review released

A new review published today in the Canadian Medical Association Journal provides guidance on Severe Combined Immunodeficiency Disease (SCID), a deadly and rare disease that is potentially curable if detected early.

18 Dec 2017

New review provides guidance to doctors who may treat infants with ‘bubble boy disease’

A new review provides guidance on a deadly, but rare, disease that is potentially curable if identified early. Severe combined immunodeficiency disease (SCID), known as the "bubble boy disease" in the 1970s, is treatable with a stem cell transplant, gene therapy and other treatments if identified at birth or soon after.

18 Dec 2017

St. Jude gene therapy offers hope for infants with 'bubble boy' disease

Early evidence suggests that gene therapy developed at St. Jude Children's Research Hospital will lead to broad protection for infants with the devastating immune disorder X-linked severe combined immunodeficiency disorder.

11 Dec 2017

Orchard Therapeutics opens new facility for technical operations in San Francisco Bay area

Orchard Therapeutics, a clinical-stage biotechnology company dedicated to transforming the lives of patients with rare disorders through innovative gene therapies, today announced the opening of a second facility for technical operations in the San Francisco Bay Area.

1 Dec 2017

Study to determine lowest dose of chemotherapy needed for babies with SCID

Michael Pulsipher, MD, of the Children's Center for Cancer and Blood Diseases at Children's Hospital Los Angeles, along with co-principal investigator, Sung-Yun Pai, MD, of Boston Children's Hospital, has been awarded nearly $9 million from the National Institute of Allergy and Infectious Diseases of the NIH to study a new treatment approach for babies born with severe combined immunodeficiency (SCID) which prevents the immune system from functioning normally.

6 Oct 2017

Severe Combined Immunodeficiency News and Research

Scientists launch clinical trial of CRISPR gene correction therapy in patients with sickle cell disease

New test will help better manage patients with severe combined immunodeficiency

Research team generates comprehensive developmental map of human T-cells

COVID-19 and immunodeficiency

NIH grants boost rare diseases research efforts

Despite failed promises, stem cell advocates again want taxpayers to pony up billions

New gene therapy cures babies with fatal 'Bubble Boy' disease

Antibody-based treatment could improve cure rate for blood and immune disorders

Leadiant Biosciences announces FDA approval of Revcovi for treating pediatric, adult ADA-SCID patients

Using novel gene therapy to cure infants born with severe combined immunodeficiency

Smart Cells launches social campaign to support Rare Disease Day 2018

A guide to Severe Combined Immunodeficiency Disease, review released

New review provides guidance to doctors who may treat infants with ‘bubble boy disease’

St. Jude gene therapy offers hope for infants with 'bubble boy' disease

Orchard Therapeutics opens new facility for technical operations in San Francisco Bay area

Study to determine lowest dose of chemotherapy needed for babies with SCID

Moms of children with rare genetic illness push for wider newborn screening

IQWiG assesses benefit of newborn screening for severe combined immunodeficiency

Researchers develop first non-human primate X-SCID models using genome editing techniques

Genome engineering-based methods pave way for new treatment of patients with sickle cell disease

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