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CGD Society - Gene Therapy film - Prof. Adrian Thrasher In this film, Prof. Adrain Thrasher of the UCL Institute of Child Health, England, talks about the progress that has been made with gene therapy over recent years

Ascendis Pharma Late-breaker oral presentation of TransCon PTH Phase 3 PaTHway Trial data in adult hypoparathyroidism on Tuesday, June 14 Oral presentation of TransCon PTH Phase 2 PaTH Forward Trial Open-Label Extension Data in adult hypoparathyroidism on Monday, June 13 PaTHway Trial results represent the second consecutive successful pivotal Phase 3 trial of a TransCon product candidate COPENHAGEN, Denmark, June 07, 2022 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced that its presentations at ENDO 2022 will include oral presentations of Phase 2 and Phase 3 data for its investigational product candidate TransCon PTH in adult hypoparathyroidism (HP). During the meeting, which will be held in-person and virtually June 11-14 in Atlanta, Dr.

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)-- Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that new data from clinical trials of 19 approved and investigational medicines across 20 cancer types will be presented at the 2021 ASCO Annual Meeting, which will be held June 4-8, 2021. A total of 132 abstracts that include a Genentech medicine will be presented at this year's meeting. These data advance oncology by showing the importance of making patient-centric treatment decisions and providing tailored medical care based on specific cancer types

At left, image shows white blood cells (red) from one of the X-CGD clinical trial participants before gene therapy. At right, after gene therapy, white blood cells from the same patient show the presence of the chemicals (blue) needed to attack and destroy bacteria and fungus.

Early Clinical Data Support ex vivo Hematopoietic Stem Cell Gene Therapy as a Potentially Promising Treatment Option for X-CGD BOSTON and LONDON, Jan. 29, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (ORTX), a global gene therapy leader, today announced that it has received orphan drug designation from the U.S

UCLA Broad Stem Cell Research Center/Nature Medicine At left, image shows white blood cells (red) from one of the X-CGD clinical trial participants before gene therapy. At right, after gene therapy, white blood cells from the same patient show the presence of the chemicals (blue) needed to attack and destroy bacteria and fungus.

'); } else { $(".fotorama-caption").addClass("remove_caption"); } }) .fotorama(); MCLEAN, United States (AFP) When a person's immune system is impaired by a genetic disease a bone marrow transplant can be a powerful therapeutic tool, but with a major downside during the first few months the recipient's defences against viruses are severely weakened. The slightest infection can lead to a hospital trip. A still-experimental type of treatment known as T-cell therapy aims to assist during this vulnerable period the months during which the body is rebuilding its natural defences

McLean (United States) (AFP) When a person's immune system is impaired by a genetic disease, a bone-marrow transplant can be a powerful therapeutic tool, but with a major downside: during the first few months the recipient's defenses against viruses are severely weakened.

Registrational Trial for Wiskott-Aldrich Syndrome Met Key Primary and Secondary Endpoints at Three Years; Data from Integrated Analysis Reinforce Treatment Benefits of Gene Therapy and Durability of Effect in Additional Patients Similar Profiles Reported Between Cryopreserved and Fresh Formulations of OTL-101, Further Supporting Upcoming Regulatory Filing and Broad Patient Availability BOSTON and LONDON, Nov. 06, 2019 (GLOBE NEWSWIRE) -- Orchard Therapeutics (ORTX), a leading commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies, today announced the upcoming presentation of registrational data from multiple programs at the 61st American Society of Hematology (ASH) Annual Meeting in Orlando, FL

The development of gene therapy vectors for the hereditary immunodeficiency Chronic Granulomatous Disease (CGD) is hampered by the absence of human cell lines, necessary for rapid and effective gene therapy vector testing. A new model supported by EU funding can make the process more efficient

FINDINGS UCLA researchers have developed a stem cell gene therapy cure for babies born with adenosine deaminase-deficient severe combined immunodeficiency, a rare and life-threatening condition that can be fatal within the first year of life if left untreated.

March 28, 2017 by Tiare Dunlap Evangelina Vaccaro (far right), who in 2012 received treatment developed by UCLAs Dr. Donald Kohn for bubble baby disease, with her family before her first day of school. Credit: Courtesy of the Vaccaro family UCLA researchers have developed a stem cell gene therapy cure for babies born with adenosine deaminase-deficient severe combined immunodeficiency, a rare and life-threatening condition that can be fatal within the first year of life if left untreated

March 1, 2017 by Marilynn Marchione This 2009 colorized microscope image made available by the Sickle Cell Foundation of Georgia via the Centers for Disease Control and Prevention shows a sickle cell, left, and normal red blood cells of a patient with sickle cell anemia. Researchers say a French teen who was given gene therapy for sickle cell disease more than two years ago now has enough properly working red blood cells to dodge the effects of the disorder. The case is detailed in the March 2, 2017 issue of the New England Journal of Medicine

1. Introduction Hematopoietic stem cell transplantation (HSCT) has a half-century history. It is currently an indispensable treatment for not only incurable blood diseases such as aplastic anemia and severe hemolytic anemia, but also malignant hematological diseases such as leukemia and lymphoma

Gene Therapy for Diseases Gene Therapy has made important medical advances in less than two decades.

Chronic Granulomatous Disease (CGD) Assignment for Gene Therapy subject. FBME, UTM

GWEN IFILL: For all of the progress made in the fight against AIDS, it still takes a terrible toll. More than 35 million people are infected with HIV around the world. More than two million people are newly infected each year

AMES, Iowa, April 4, 2013 /PRNewswire/ --NewLink Genetics Corporation (NLNK) is a biopharmaceutical company focused on discovering, developing and commercializing cancer therapeutics.

ELK GROVE VILLAGE, Ill., March 13, 2013 (GLOBE NEWSWIRE) -- Nationwide Children's Hospital (Columbus, OH) and Families of Spinal Muscular Atrophy (Elk Grove Village, IL) announce the award of a multi-million dollar cooperative agreement from the National Institute of Neurological Disorders and Stroke (NINDS) to advance a gene therapy development program for Spinal Muscular Atrophy (SMA). This three-year multi-million dollar cooperative agreement to Brian Kaspar, PhD, principal investigator in the Center for Gene Therapy at The Research Institute at Nationwide Children's Hospital in the amount of $3,752,462, funds pre-clinical drug development up to the filing of an Investigational New Drug Application (IND) to the Food and Drug Administration (FDA).

29 February 2012 Last updated at 10:39 ET Treatment which aims to correct a rare inherited genetic defect has helped a patient at risk from serious infection, a leading hospital is reporting. The use of gene therapy against chronic granulomatous disorder (CGD) is a third success for Great Ormond Street Hospital in London. Patients with CGD cannot make cells to fight bacterial and fungal infection.

Image source: The Motley Fool. Orchard Therapeutics(NASDAQ:ORTX)Q12020 Earnings CallMay 9, 2020, 8:30 p.m. ET Operator Ladies and gentlemen, thank you for standing by, and welcome to the Orchard Therapeutics First Quarter 2020 Investor Conference Call

Orchard Therapeutics plc (NASDAQ:ORTX) Q1 2020 Earnings Conference Call May 7, 2020 8:00 AM ET Company Participants Renee Leck Director-Investor Relations Bobby Gaspar Chief Executive Officer Frank Thomas Chief Operating Officer Conference Call Participants Whitney Ijem Guggenheim Esther Rajavelu Oppenheimer Yaron Werber Cowen Peter Kim Barclays Operator Ladies and gentlemen, thank you for standing by, and welcome to the Orchard Therapeutics First Quarter 2020 Investor Conference Call.

Image source: The Motley Fool.

BOSTON and LONDON, Dec. 08, 2019 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a leading commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies, will be presenting new registrational data from multiple programs at the 61st American Society of Hematology (ASH) Annual Meeting being held December 7-10, 2019 in Orlando, FL.

BOSTON and LONDON, Oct. 22, 2019 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a leading commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies, today announced initial results from a clinical trial with a cryopreserved formulation of OTL-200, a gene therapy in development for the treatment of metachromatic leukodystrophy (MLD) at the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. The initial data show that cellular engraftment with OTL-200 using a cryopreserved formulation is similar to that observed using a fresh formulation with the longest patient having 12 months of follow-up since treatment