Pharmaceutical Technology

OCTOBER 25, 2022

There are options in the future to possibly apply the worldwide research and development (R&D), manufacturing and marketing expertise of Astellas in gene therapy to AAV gene therapy development programmes of Taysha for genetic ailments of the central nervous system (CNS).

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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.  She said: “Payment options will be devised to absorb the costs. One

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Gene Therapy Manufacturing Gene Clinical Trials 71

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. How Can Study Protocols Be More Effective?

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Gene Therapy Gene Trials Clinical Trials 95

XTalks

DECEMBER 29, 2022

This highlights potentially new marketing trends that life science companies may have to start looking into if they haven’t already. In 2022 alone, the US regulator approved four new gene therapies, showing the high interest in getting these therapies to market. 4. Social Media Marketing.

Life Science 52

Life Science Gene Therapy Gene Manufacturing 52

The Pharma Data

NOVEMBER 2, 2020

This acceptance by the FDA marks the first marketing application accepted for a treatment for achondroplasia in the United States. This information was included in the marketing application. BioMarin previously announced that the European Medicines Agency (EMA) validated the Company’s Marketing Authorization Application.

Drugs 40

Drugs Containment Trials Marketing 40

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. Thiola (tiopronin) is produced by Retrophin, the rare disease biopharmaceutical company founded by Martin Shkreli.

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Trials Gene Hormones Clinical Trials 98

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