Cell and Gene Therapy
THE LATEST
The rare disease drugmaker is facing potential competitors for achondroplasia drug Voxzogo. Is a big M&A deal with two approved assets enough to maintain investor interest?
A recurring theme Tuesday morning at Phacilitate’s Advanced Therapies Week was the quickly emerging potential of in vivo approaches to cell and gene therapy—a trend also reflected in recent investments by Eli Lilly and Regeneron.
A lawsuit and FDA warning ensued after Hims & Hers launched a compounded version of Novo Nordisk’s new obesity pill, more Big Pharma report earnings—including from weight loss rivals Novo and Eli Lilly—and the gene therapy space sees another rejection.
The FDA recommended that REGENXBIO run a new study, treat more patients and include a placebo arm to support a resubmission for the gene therapy RGX-121.
The deal gets Lilly access to Orna’s in vivo CAR T technology. The biotech’s lead asset, which has yet to start clinical testing, is focused on B cell–driven autoimmune diseases.
The gene therapy uses an AAV vector to restore healthy levels of the alpha-galactosidase enzyme, which is rendered dysfunctional in patients with Fabry disease, leading to the toxic build-up of lipids in cells.
The FDA in July last year declined to approve UX111 for Sanfilippo syndrome, a rare neurodegenerative disorder, citing manufacturing issues. Ultragenyx Pharmaceutical resubmitted its application ten days ago, expecting a six month review time.
The SPAC agreement values PrimeGen US at $1.5 billion in equity and gives it capital to advance its pre-clinical triple-activated mesenchymal stem cell pipeline into the clinic.
After a series of deaths in patients taking Sarepta Therapeutics’ gene therapies, doubt has crept into investor sentiments around the long-time Wall Street darling, and patients may soon begin looking elsewhere.
Investor enthusiasm and evolving FDA pathways are accelerating rare disease drug development, with ultrarare conditions like MPS II moving into the spotlight.