Pharmaceutical Technology

OCTOBER 25, 2022

Additionally, Astellas will obtain an exclusive option for licencing two clinical-stage programmes of Taysha, namely TSHA-102 and TSHA-120, for Rett syndrome and GAN, respectively. Astellas will also receive specific rights linked to any possible change of Taysha’s control.

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Gene Therapy Gene Development Genetics 264

XTalks

APRIL 24, 2023

Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday.

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Gene Therapy Gene Trials Clinical Trials 95

Drug Discovery World

JANUARY 30, 2024

“Dr Naldini open new doors for cures for otherwise untreatable genetic diseases with the full impact of his work still untapped”, she said. Intellectual property: High patent royalties and licensing fees for key technologies can inflate treatment costs. Fostering open innovation and collaboration could help address this issue.

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Gene Therapy Manufacturing Gene Clinical Trials 71

XTalks

DECEMBER 29, 2022

In 2022 alone, the US regulator approved four new gene therapies, showing the high interest in getting these therapies to market. It has also been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the US regulator for both SCD and TDT.

Life Science 52

Life Science Gene Therapy Gene Manufacturing 52

The Pharma Data

AUGUST 3, 2021

Food and Drug Administration (FDA) has determined that OAV-101 intrathecal (IT) clinical trials for spinal muscular atrophy (SMA) patients may proceed, thereby lifting the partial clinical trial hold initiated in October 2019. Novartis today announced that the U.S. SVP, Chief Medical Officer, Novartis Gene Therapies. “We

Clinical Trials 52

Clinical Trials Clinical Trials Gene Therapy Trials 52

XTalks

SEPTEMBER 17, 2020

In rare disease trials, it’s not always feasible to choose clinically-relevant endpoints to measure the efficacy of a new therapeutic. Patients with this disease have a very high level of LDL-C, which increases their risk of coronary artery disease, including heart attack, stroke and atherosclerosis.

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Trials Gene Hormones Clinical Trials 98

The Pharma Data

NOVEMBER 2, 2020

We are grateful to the families who have participated in the clinical trials, the advocacy groups from around the world, and the study investigators, all who have been on this journey with us to deliver a treatment choice and contribute to further understanding achondroplasia medically and scientifically.” In the U.S.,

Drugs 40

Drugs Containment Trials Marketing 40