The Woleben family: Lauren, Doug, Will and Kasey (left to right). The family is eager to see a treatment for Will's ultra-rare disease. Courtesy photo

A biotech paused a gene ther­a­py hatched by fam­i­lies. Now they want con­trol

Will Woleben is like many 10-year-olds. He loves to watch sports and play jokes on his lit­tle sis­ter. But Will can no longer walk, among oth­er abil­i­ties he lost. If noth­ing is done, he will con­tin­ue to de­gen­er­ate and then die at a young age.

His par­ents — and oth­er fam­i­lies af­fect­ed by the ul­tra-rare con­di­tion Leigh syn­drome — formed the Cure Mi­to Foun­da­tion and raised $1 mil­lion to spark a gene ther­a­py. The com­pa­ny Taysha Gene Ther­a­pies ad­vanced the drug pro­gram. But in March, Taysha paused de­vel­op­ment.

Endpoints News

Unlock this article instantly by becoming a free subscriber.

You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.