A mother is taking an innovative approach to raise money for a gene therapy treatment for her 7-year-old son’s rare genetic condition. Amber Freed hopes to secure $1 million to fund the manufacturing of the treatment and early trials, offering to name the treatment after the highest-bidding donor.
The effort comes as the biotech industry faces challenges in developing treatments for rare diseases due to limited resources and commercial interest. Gene therapy, in theory, holds promise for treating various conditions, but its applications are often hampered by rarity and lack of funding.
Meanwhile, other companies announced notable developments:
* Edgewise Therapeutics reported positive results from a study involving an experimental drug designed to prevent muscle damage in Duchenne muscular dystrophy patients.
* Neurocrine Biosciences won FDA approval for a treatment for classic congenital adrenal hyperplasia, a rare disease affecting the adrenal glands.
* Abbvie acquired Nimble Therapeutics for $200 million, expanding its portfolio of autoimmune disease treatments.
Viridian Therapeutics confirmed benefits from an experimental treatment for thyroid eye disease, with 56% of participants showing significant improvement. The company plans to include results in its marketing application to the FDA.
Regulatory agencies also made announcements:
* The FTC declined to challenge Novo Holdings’ acquisition of Catalent, a contract pharmaceutical manufacturer.
* South African regulators concluded that Vertex Pharmaceuticals is offering sufficient access to its cystic fibrosis treatment Trikafta.
Source: https://www.statnews.com/2024/12/16/biotech-news-viridian-edgewise-therapeutics-vertex-novo-catalent-cystic-fibrosis-the-readout