Gene therapy trial gives deaf children hearing in both ears;
Study participants born unable to hear could locate sound sources, recognise speech and dance to music after treatment
https://www.theguardian.com/science/article/2024/jun/05/gene-therapy-trial-restores-hearing-in-both-ears-for-deaf-children
#science #news #biology #deafness #GeneTherapies #genetics #breakthrough
Gene therapy trial gives deaf children hearing in both ears

Study participants born unable to hear could locate sound sources, recognise speech and dance to music after treatment

The Guardian
Lilly trumpets early success with deafness gene therapy

Eli Lilly’s gene therapy for otoferlin-related deafness has restored the hearing of the first child enrolled in its ongoing phase 1/2 trial.

pharmaphorum

NSW Health, Australia, has set a national benchmark for standardising the preparation of pharmaceuticals and advanced therapeutic products with a new Policy Directive that outlines new and updated state-wide approaches.

Learn more about the Directive & author Kerry Watts: https://www.medicalresearch.nsw.gov.au/future-proofing-nsw-health-production-ofpharmaceuticals-and-advanced-therapeutics/ #AdvancedTherapies #CellTherapies #GeneTherapies

German researchers welcome national, EU support for gene therapy research

National and EU-level support goes a long way towards supporting research into gene therapies against rare dis

EURACTIV
Greek legal framework fails to address complexities of access to gene therapies

The Greek legislative framework lacks concrete solutions to advance gene therapies and improve access for pati

EURACTIV
Italy’s advanced therapy journey

Advanced Medicinal Therapies (ATMPs), including gene and cell therapies, offer treatment options for patients

EURACTIV
EU needs to step up to unleash gene therapies potential, stakeholders say

While gene therapies have the potential to transform the approach to rare diseases and improve the quality of

EURACTIV
AAPS Statement Calling for Moratorium on COVID-19 Shot Mandates and Genetic Injections - AAPS | Association of American Physicians and Surgeons

1.  COVID 19 injections are under Emergency Use Authorization and must be considered experimental.  Informed consent is a bedrock principle of medical ethics, yet millions of people have taken COVID-19 injections under duress. 2.  The […]

AAPS | Association of American Physicians and Surgeons

#GeneTherapies for rare diseases are under threat. Scientists hope to save them.

https://www.nature.com/articles/d41586-023-03109-z?utm_source=Nature+Briefing&utm_campaign=92a97c1689-briefing-dy-20231009&utm_medium=email&utm_term=0_c9dfd39373-92a97c1689-46206854

"The Italian charity Telethon Foundation in Milan has announced that it will produce #Strimvelis itself. “We are making a paradigm shift,” said Francesca Pasinelli, general manager of the Telethon Foundation, in a 12 September statement announcing the decision. “We are the first non-profit organization to take on the commercialization of a gene therapy.”

Gene therapies for rare diseases are under threat. Scientists hope to save them

As industry steps aside, scientists seek innovative ways to make sure expensive treatments can reach people who need them.

AAAS: "Hello kitty, goodbye kittens? Gene therapy spays cats without surgery." Spaying or neutering cats + dogs has the added benefit of protecting against mammary cancer + other disorders. Globally, more than half of the approimately 1.5 billion dogs + cats are homeless. "Many die on the streets, victims of cars or disease; others are killed in mass culling campaigns that seek to protect wildlife or prevent the spread of rabies." Spay/neuter surgeries casn help, but large-scle sterilization of often feral animals is infeasible. 'David Pépin, a reproductive biologist at Massachusetts General Hospital, spent his early career researching antimüllerian hormone (AMH), which is produced by follicles in the ovary that give rise to eggs.' Pépin + colleagues inserted the AMH gene with a harmless virus often used to ferry replacement genes into cells with an injection into thigh muscles. The treated cats failed to ovulate, while the sham-injected controls remained fertile. One cat followed over 5 yrs continued to exhibit elevated AMH. A meeting is planned next month with the U.S. Food and Drug Administration to map out larger safety + efficacy studies, but a commercial product will probably not be available for 5 yrs. Having far fewer unsupervised cats in the world would protect many birds + ecosystems. #spayandneuter #genetherapies #overpopulation