World's First Patient to Receive Moderna's mRNA Therapy for Genetic Disease

Scientists first identified and recognized the importance of mRNA in 1960, and it has since been studied and explored for use in vaccines and disease therapies. But until the end of 2020, the two mRNA COVID-19 vaccines were approved for marketing. In the past 60 years, most people have never heard of mRNA vaccines or mRNA drugs.

Prior to the COVID-19 pandemic, Moderna was researching the use of mRNA to treat glycogen storage disease type Ia (GSD-Ia), a severe genetic metabolic disease in which patients acquire glucose- 6-phosphatase inactivation, resulting in low blood sugar in the body. In addition, it is accompanied by a series of clinical symptoms such as hepatomegaly, acidosis, hyperlipidemia, hyperuricemia, hyperlactatemia, coagulation dysfunction, developmental delay, and other life-threatening conditions.

Loveah Hernandez of Dallas, Texas, was one such unfortunate youngster who was diagnosed with GSD-Ia in the hospital at the age of 6 months when she suddenly exhibited unresponsive symptoms.

Her life has been challenging since then in order for her to survive - a feeding tube is installed from her nose to her stomach so that she can acquire the blood sugar level she requires through nasal feeding of cornstarch that can pass through the intestines Because cornstarch can stay in the gut for a long time and release glucose slowly. At age 3, the tube was replaced with a gastrostomy tube through which cornstarch was supplemented for nutrition. She, like other patients with GSD-Ia, must take cornstarch every 3-4 hours to keep alive. This meant she'd never be able to sleep a single night, and her mother had to go to school on time every day to ensure her study and safety.

Loveah Hernandez has worked hard to become a trial participant after learning that Moderna was initiating a clinical study of an mRNA treatment for GSD-Ia. She has spent the past two years making sure she is eligible to participate in the clinical trial.

The new therapy, known as mRNA-3745, is designed to test the safety of mRNA in patients with GSD-Ia and to see if it can help fix the deficiency that causes GSD-Ia and restore normal function in patients. If the trial is successful, mRNA-3745 will help liver cells effectively break down glycogen, relieve hypoglycemic symptoms, and reduce the requirement for patients to take starch on a regular basis.

Moderna and UConn Children's Hospital began planning for this clinical research four years ago, and it officially began in July 2021. After a year of planning and preparation, Loveah Hernandez became the world's first patient to receive mRNA treatment for GSD-Ia, receiving a lipid nanoparticle (LNP)-delivered mRNA for the G6PC gene through an intravenous infusion in her arm. "It was incredibly amazing to be the first to receive this exploratory treatment," stated Loveah Hernandez.

The clinical trial's chief physician, Juan C. Salazar, of the University of Connecticut Children's Hospital, said, "this clinical trial is exciting and historic, and the mRNA vaccines for COVID-19 allowed us to realize the effectiveness of mRNA, and now we're leveraging the same technology platform to treat a completely different disease, which might be a game changer for children and their families all around the world."