REGENERATIVE MEDICINE – Page 7 – REGENHEALTHSOLUTIONS (RHS)

PIER MARIA FORNASARI
2022-11-10

New Exa-cel gene therapy for Sickle Cell Disease and Beta Thalassemia global regulatory submission in 2022

A potential 1-time gene editing treatment for severe sickle cell disease (SCD) and transfusion-dependent β-thalassemia (TDT) is entering approval review by the US Food and Drug Administration (FDA), the European Medicines Agency, and the UK Medicines and Healthcare products Regulatory Agency, according to a company statement. Both diseases involve variants in the gene encoding β globin. The variants

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PIER MARIA FORNASARI
2022-11-09

US FDA grants Rare Pediatric Disease Designation to gene therapy OTOF-GT for the treatment of otoferlin gene-mediated hearing loss

OTOF-GT targets the restoration of hearing in people living with otoferlin deficiency Sensorion, a pioneering clinical-stage biotechnology company which specializes in the development of novel therapies to restore, treat and prevent hearing loss disorders, announces that the US Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to the Company’s lead therapy gene

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PIER MARIA FORNASARI
2022-11-07

Novel CRISPR/Cas9-based gene-editing therapy shows promise for patients with transthyretin amyloid cardiomyopathy

A single IV infusion of NTLA-2001, a novel CRISPR/Cas9-based gene editing therapy, significantly reduced circulating transthyretin (TTR) protein levels in patients with ATTR amyloid cardiomyopathy, a progressive and fatal cause of heart failure, according to late-breaking research presented today at the American Heart Association’s Scientific Sessions 2022. The meeting, held in person in Chicago and

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PIER MARIA FORNASARI
2022-11-07

First ever clinical trial of lab-grown red blood cell transfusion

Cambridge researchers are taking part in the world’s first clinical trial of red blood cells that have been grown in a laboratory for transfusion into another person. The manufactured blood cells were grown from stem cells from donors. The red cells were then transfused into volunteers in the RESTORE randomised controlled clinical trial. This is

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PIER MARIA FORNASARI
2022-11-03

Rehabilitation combined with neural progenitor cell grafts enables functional recovery in chronic spinal cord injury

In recent years, researchers have made measurable progress, using animal models, to promote tissue regeneration in spinal cord injuries (SCI) through implanted neural stem cells or grafts. Other efforts have shown that intensive physical rehabilitation can improve function after SCI by promoting greater or new roles for undamaged or spared cells and neural circuits. In

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PIER MARIA FORNASARI
2022-11-03

CAR-T cells therapy for autoimmune diseases

A recent study published in the journal Nature Medicine highlights the potential of CAR T therapy beyond this realm—specifically for lupus and other autoimmune diseases. What is Lupus? Lupus (systemic lupus erythematosus) is an autoimmune disease that affects women approximately ten more than men, and is characterized by the overproduction of antibodies that attack the body’s own tissues. Lupus

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PIER MARIA FORNASARI
2022-10-23

Drug Resistant Multiple Myeloma and CAR-T cells therapy

Multiple myeloma is a relatively uncommon yet serious disease estimated to impact more than 30,000 US citizens this year. Although several treatment options exist, the illness is considered incurable as most treatments do not resolve the condition permanently—including the most recent advancements with CAR T cells. Here, we describe an approach using a different variant

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PIER MARIA FORNASARI
2022-10-17

First AAV gene therapy approved in Europe for direct infusion into the brain as a treatment for AADC Deficiency.

Gene Therapy Sees Children With Rare Genetic Condition Walk And Talk For First Time Aromatic l-amino acid decarboxylase (AADC) deficiency is a genetic disorder of the nervous system. Only about 120 cases have ever been reported, and around half of these have been in people of Asian ancestry. The condition is caused by mutations in the DDC gene, which

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PIER MARIA FORNASARI
2022-10-16

New research on CAR-T cells clinical applications

One of the dreams of cancer therapy is to use the power of the body to heal itself. This dream, long in the making, is becoming a reality thanks to deep and fundamental understandings of the immune system, the primary means by which we protect ourselves from external and internal threats. The immune system recognizes

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PIER MARIA FORNASARI
2022-10-14

First stem cell treatment for spina bifida delivered during fetal surgery performed at UC Davis Health

Groundbreaking trial aims to reverse the paralysis and other abnormal functions of spina bifida before birth Three babies have been born after receiving the world’s first spina bifida treatment combining surgery with stem cells. This was made possible by a landmark clinical trial at UC Davis Health. The one-of-a-kind treatment, delivered while a fetus is

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REGENERATIVE AND PERSONALISED MEDICINE

DIGITAL HEALTHCARE

Category REGENERATIVE MEDICINE

New Exa-cel gene therapy for Sickle Cell Disease and Beta Thalassemia global regulatory submission in 2022

US FDA grants Rare Pediatric Disease Designation to gene therapy OTOF-GT for the treatment of otoferlin gene-mediated hearing loss

Novel CRISPR/Cas9-based gene-editing therapy shows promise for patients with transthyretin amyloid cardiomyopathy

First ever clinical trial of lab-grown red blood cell transfusion

Rehabilitation combined with neural progenitor cell grafts enables functional recovery in chronic spinal cord injury

CAR-T cells therapy for autoimmune diseases

Drug Resistant Multiple Myeloma and CAR-T cells therapy

First AAV gene therapy approved in Europe for direct infusion into the brain as a treatment for AADC Deficiency.

New research on CAR-T cells clinical applications

First stem cell treatment for spina bifida delivered during fetal surgery performed at UC Davis Health