In the News

Southport Mum renews appeal for daughter with rare disorder

Submitted by Sarah Steel Having been featured in the Liverpool ECHO last year, Sarah Steel, a mother from Southport whose daughter, Isla, suffers from a rare metabolic disorder, is now renewing her daughter’s appeal in an effort to generate funding for scientists to explore gene therapy trials. In 2018,

Mesa family hosting virtual walkathon for NKH awareness

MESA, AZ (3TV/CBS 5) – A Mesa family is hosting a “Zoom the Room” virtual walkathon to raise awareness for NKH and money for gene replacement therapy research, which could be life-saving for their little boy. Click to Watch video of the story on AZ Family. Lucas Launched for Fundraising for Rare Disease Video Conferencing Lucas John Foundation is Actively Working to Save Children with Orphaned Rare Diseases FOR IMMEDIATE RELEASE   May 2020 (Mesa, Arizona) – The family of Brandon and Chancee Culp in Mesa, Arizona, is fighting for the life of their two-year-old son, Lucas John Culp.

Rare disease is Not so Rare. Rare diseases in the U.S. Affect 25–30 million

Rare diseases are by their very nature obscure and unknown, falling through the cracks when it comes to research budgets and treatment development. But now, a study has shown they are more common than we think. New research shows that rare diseases are not that rare. The

Gene Therapy AAV-based COVID-19 vaccine

Luk Vandenberghe, a gene therapy researcher at Massachusetts Eye and Ear and at Harvard Medical School, is spearheading an effort to develop a COVID-19 vaccine that uses an adeno-associated viral (AAV) vector to deliver genetic instructions for the SARS-CoV-2 spike protein into human cells. AAV vectors are

Patient Trial of Gene Therapy

1st Patient Dosed in Trial of Gene Therapy, OTL-201, for Sanfilippo Type A Orchard Therapeutics announced the dosing of a first patient in a clinical trial evaluating its investigational cell-based gene therapy OTL-201 for Sanfilippo syndrome type A. OTL-201’s safety, tolerability, and efficacy is being investigated in the open-label, Phase 1/2 study (NCT04201405)

CEO chosen for new Nationwide Children’s gene-therapy company

Nationwide Children’s Hospital chooses Mayo Pujols, most recently of Novartis Pharmaceuticals, for its new biotech subsidiary Andelyn. As the new CEO for Andelyn Biosciences, a Nationwide Children’s Hospital-owned gene-therapy startup, Mayo Pujols will begin to assemble a diverse team of up to 200 scientists, biochemists, engineers and

With $30 million in funding, Dallas startup will speed gene therapy clinical trials

A new biotech startup in Dallas aims to speed the development of treatments for rare neurological diseases that disproportionately affect children. Taysha Gene Therapies, launched Tuesday with $30 million in seed financing, has partnered with UT Southwestern researchers whose mission is to eradicate genetic diseases that can

McDonald’s staff tackle Snowdon challenge for Isla

A group of 20 individuals from Kew McDonalds in Southport have volunteered to complete a sponsored Snowdon climb on the 30th of October 2019. We are fundraising for the beautiful Isla Rose and the John Lucas Foundation. Isla was born 30th September 2018. After a wonderful pregnancy

Infant son’s rare genetic disease inspire Mesa couple

Twenty-four hours after the birth of their son, a Mesa couple’s lives were turned upside down after learning he was born with a rare medical condition. For weeks, in between hospital stays and new challenges, Brandon and Chancee Culp never gave up hope and now they’re raising