Drug Discovery World

JANUARY 30, 2024

Viral vector manufacturing At the Advanced Therapies Awards ceremony, Dr Luigi Naldini, Director of the San Raffaele Telethon Institute for Gene Therapy, was awarded a Lifetime Achievement Award in recognition of ‘extraordinary contributions to gene therapy’, a field he has helped shape through his pioneering work on lentiviral vectors.

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Gene Therapy Manufacturing Gene Clinical Trials 71

XTalks

DECEMBER 29, 2022

For example, on the pharma and biotech side, there has been growing demand for new drugs and biologics, from new classes of dual-action diabetes and weight loss drugs like Ozempic and Mounjaro to life-saving gene therapies. 1. Cell and Gene Therapies. In 2023, a number of gene therapies are expected to get the FDA green light.

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Life Science Gene Therapy Gene Manufacturing 52

Drug Discovery World

MARCH 24, 2023

The research team from Ben-Gurion University of the Negev and Soroka Medical Center discovered the severe hereditary muscle disease that develops around the age of 40 years, progressing to complete immobility of the limbs and the chest muscles.

Research 52

Research Genetics Gene Licensing 52

The Pharma Data

NOVEMBER 29, 2020

Y-mAbs Therapeutics has a target action date of November 30 for its Biologics License Application (BLA) for Danyelza (naxitamab) for patients with relapsed/refractory high-risk neuroblastoma. The drug was developed by researchers at Memorial Sloan Kettering Cancer Center and exclusively licensed to Y-mAbs.

Genetic Disease 52

Genetic Disease Antibody Licensing Licensing 52

The Pharma Data

DECEMBER 20, 2020

Athenex and Almirall have a license deal with Almirall having the license to research, develop and commercialize the drug in the U.S. CF is a rare, life-shortening genetic disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract. and Europe, including Russia.

In-Vitro 52

In-Vitro FDA Approval Manufacturing Drugs 52

XTalks

DECEMBER 13, 2023

The year 2022 has proven to be a momentous period for Fulgent Genetics, marked by significant expansion of its product portfolio across various medical conditions. Xoma’s unique approach involves acquiring future economic interests tied to pre-commercial therapeutic candidates that have been licensed to pharmaceutical or biotech companies.

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Genetics Vaccination Vaccine DNA 111

The Pharma Data

JANUARY 17, 2021

Based in Seattle, Washington, Sana focuses on in vivo and ex vivo cell engineering platforms to develop therapies for cancer, diabetes, cardiovascular disease, CNS disorders, and genetic diseases.

In-Vivo 52

In-Vivo Marketing Engineer Genetics 52

pharmaphorum

DECEMBER 8, 2021

Rett syndrome is caused by mutations in the MECP2 gene found on the X chromosome and leads to problems in brain function that cause behavioural problems, a rapid decline in the ability to speak and carry out manual tasks, as well as seizures, curvature of the spine and sleep disturbances.

Marketing 59

Marketing Drugs Life Science Trials 59

The Pharma Data

NOVEMBER 8, 2020

Bristol Myers Squibb has a target action date of November 16 for the biologics license application (BLA) for lisocabtagene maraleucel (liso-cel), a CD19-directed CAR T cell therapy for adults with r/r large B-cell lymphoma after at least two previous therapies.

Trials 52

Trials Protein Gene Genetic Disease 52

XTalks

SEPTEMBER 17, 2020

Inherited mutations in the SLC3A1 and SLC7A9 genes cause cystine to be abnormally transported within the kidney leading to cystinuria. Symptoms and Etiology: Characterized by progressive muscle weakness and atrophy, Duchenne muscular dystrophy (DMD) is an X-linked genetic condition that primarily affects males. galactosidase A (?-Gal

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Trials Gene Hormones Clinical Trials 98

The Pharma Data

NOVEMBER 11, 2020

Food and Drug Administration (FDA) for INZ-701 for the treatment of ENPP1 deficiency – The FDA grants rare pediatric disease designation to drugs for serious and life-threatening diseases in which the serious or life-threatening manifestations primarily affect children aged from birth through 18 years and affect fewer than 200,000 people in the U.S.

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Clinical Trials Clinical Trials Trials Development 40

The Pharma Data

NOVEMBER 2, 2020

This condition is caused by a mutation in the fibroblast growth factor receptor 3 gene ( FGFR3 ), a negative regulator of bone growth. More than 80% of children with achondroplasia have parents of average stature and have the condition as the result of a spontaneous gene mutation. In the U.S., About BioMarin.

Drugs 40

Drugs Containment Trials Marketing 40

The Pharma Data

AUGUST 3, 2021

SVP, Chief Medical Officer, Novartis Gene Therapies. “We Additionally, STEER will add to the clinical data and emerging real-world evidence for the use of gene therapy to treat SMA. This route of administration has the potential to open up access for older patients to all the benefits of gene therapy.

Clinical Trials 52

Clinical Trials Clinical Trials Gene Therapy Trials 52

Drug Discovery World

MARCH 6, 2024

Guiding neurological drug discovery and development to success The 20 years since the Human Genome Project has seen transformational advances in the molecular understanding of cancers and rare genetic diseases, leading to genetically informed, personalised selection of therapies and massively improved outcomes for many patients.

Genome 64

Genome Genomics Clinical Trials Clinical Trials 64

The Pharma Data

OCTOBER 15, 2020

Axovant – Kristin Vuori was named to the board of directors at Axovant Gene Therapies Ltd. Sabbagh will be responsible for expanding Inozyme’s proprietary pipeline by identifying and developing new therapeutics for monogenic and non-genetic diseases of abnormal mineralization. Additionally, Parag V.

In-Vivo 52

In-Vivo Business Development Manufacturing Development 52

The Pharma Data

OCTOBER 28, 2020

Five patients between the ages of 5 and 15 with deletions in the maternal UBE3A gene region were enrolled in the first three cohorts and are included in the interim data analysis. Angelman syndrome is a rare, neurogenetic disorder caused by loss-of-function of the maternally inherited allele of the UBE3A gene. About Angelman Syndrome.

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Protein Production Development Drugs 40

Drug Discovery World

FEBRUARY 22, 2024

For diseases as serious and debilitating for patients and their families as leukodystrophies, learning about such advances in knowledge carries a great deal of hope, which IRCM warmly welcomes,” adds Dr Jean-François Côté, the IRCM’s President and Scientific Director.

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Drugs Gene Clinical Trials Clinical Trials 59