REGENERATIVE MEDICINE – REGENHEALTHSOLUTIONS (RHS)

PIER MARIA FORNASARI
2024-10-14

Researchers at Children’s Hospital of Philadelphia Reveal New Insights into Non-Inflammatory Causes of Rare Neurological Symptoms in Children, Adolescents and Young Adults Following CAR-T Therapy

Study marks the first time these side effects were reported in children Researchers at Children’s Hospital of Philadelphia (CHOP) revealed for the first time that children, adolescents and young adults may experience very rare neurological issues of paraparesis and quadriparesis following chimeric antigen receptor T-cell (CAR-T) therapy, a type of immunotherapy used to treat B-cell Acute

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PIER MARIA FORNASARI
2024-09-26

New Perspectives on the Risk of Secondary Cancers After CAR T-cell Therapy

When the U.S. Food and Drug Administration (FDA) announced in November 2023 that it was investigating a link between receiving CAR T therapy and developing secondary cancers later on, it spurred uncertainty for patients and researchers alike. As many of the more than 30,000 patients who have received the life-saving treatment face new questions about

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PIER MARIA FORNASARI
2024-09-26

Single-Dose Gene Therapy is Potentially Life-Changing for Adults with Hemophilia B

Study results formed the basis of the therapy’s FDA approval in April 2024 Adults with hemophilia B saw their number of bleeding episodes drop by an average of 71 percent after a single infusion of gene therapy, according to the results of an international Phase III clinical trial published today in the New England Journal of Medicine by researchers from the University of

No Comments InGENE THERAPY RARE DISEASES

PIER MARIA FORNASARI
2024-09-26

Cells programmed to target brain tumors

Scientists from UNIGE and HUG have developed CAR-T cells capable of targeting malignant gliomas while preserving healthy tissues. Glioblastoma is the most common and aggressive primary brain tumor—average survival after diagnosis is less than two years—and current treatments remain ineffective. In recent years, immunotherapies have been giving patients hope, albeit with relatively modest success. A

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PIER MARIA FORNASARI
2024-07-11

Trial of cell-based therapy for high-risk lymphoma leads to FDA breakthrough designation

In an early Stanford Medicine study, CAR-T cell therapy helps some with intractable lymphoma, but those who relapse have few options. Modifying the therapy’s molecular target improved response. CAR-T cell therapy, which targets a specific protein on the surface of cancer cells, causes tumors to shrink or disappear in about half of patients with large

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PIER MARIA FORNASARI
2024-06-22

Risk of secondary cancers after CAR-T cell therapy low, according to large Stanford Medicine study

In April, the FDA warned of risk of secondary cancers in people receiving CAR-T cell therapy. A large Stanford Medicine study finds the risk is low and not related to the CAR-T cells. A large study by researchers at Stanford Medicine has found that the risk of secondary blood cancers after CAR-T cell therapy — a

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PIER MARIA FORNASARI
2024-06-07

New Gene Therapy Trial Shows Restored Hearing and Speech in Children Born Deaf, Treated in Both Ears

Administering gene therapy in both ears of five children with DFNB9 led to additional benefits compared to previous trial, including sound source localization and hearing in noisy environments. Key Takeaways A novel gene therapy designed to target a form of inherited deafness restored hearing function in five children who were treated in both ears. The

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PIER MARIA FORNASARI
2024-05-11

Regeneron Presents Update on Gene Therapy for Genetic Deafness at ASGCT

Regeneron presented results from an ongoing Phase I/II clinical trial for its investigational gene therapy, DB-OTO, at the annual American Society of Gene and Cell Therapy (ASGCT) meeting, being held in Baltimore, Md. from May 7–11. DB-OTO, a gene therapy for genetic deafness, improved hearing in one child, treated at 11 months old to normal levels within

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PIER MARIA FORNASARI
2024-05-11

Gene Editing Improves Vision in Some People With Inherited Blindness

First-of-its-kind trial suggests CRISPR acting inside the body can be safe and effective At a glance: Results from a small proof-of-concept study indicate that CRISPR gene editing is safe and can improve vision in some people with inherited blindness. In the multicenter clinical trial, 11 of the 14 individuals treated had measurable improvements on at

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PIER MARIA FORNASARI
2024-05-11

Fred Hutch scientist Dr. Adair partners with colleagues around the globe to make gene therapies more effective and more widely available

Fred Hutch Cancer Center scientist Jennifer E. Adair, PhD, is on a mission to foster worldwide collaboration on potentially curative gene therapies. Holder of the Fleischauer Family Endowed Chair in Gene Therapy Translation, Adair just co-authored two articles published today in Science Translational Medicine as part of a special series on global access to these therapies that she

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REGENERATIVE AND PERSONALISED MEDICINE

DIGITAL HEALTHCARE

Category REGENERATIVE MEDICINE

Researchers at Children’s Hospital of Philadelphia Reveal New Insights into Non-Inflammatory Causes of Rare Neurological Symptoms in Children, Adolescents and Young Adults Following CAR-T Therapy

New Perspectives on the Risk of Secondary Cancers After CAR T-cell Therapy

Single-Dose Gene Therapy is Potentially Life-Changing for Adults with Hemophilia B

Cells programmed to target brain tumors

Trial of cell-based therapy for high-risk lymphoma leads to FDA breakthrough designation

Risk of secondary cancers after CAR-T cell therapy low, according to large Stanford Medicine study

New Gene Therapy Trial Shows Restored Hearing and Speech in Children Born Deaf, Treated in Both Ears

Regeneron Presents Update on Gene Therapy for Genetic Deafness at ASGCT

Gene Editing Improves Vision in Some People With Inherited Blindness

Fred Hutch scientist Dr. Adair partners with colleagues around the globe to make gene therapies more effective and more widely available