Iecure plans to file a clinical trial application as early as 2022 to advance Precision's PBGENE-PCSK9 clinical candidate through phase I studies for the treatment of familial hypercholesterolemia (FH), the companies said.

Precision will retain rights to PBGENE-PCSK9, including products developed for genetic indications with increased risk of severe cardiovascular events such as FH. In return, Precision has granted Iecure a license to use its PCSK9-directed Arcus nuclease to insert genes into the PCSK9 locus to develop treatments for four other prespecified, rare genetic diseases.

Precision will receive an equity stake in Iecure and is eligible to receive milestone and royalty payments on sales of Iecure products developed with Arcus.

In a separate announcement, Iecure said that it had raised $50 million in a financing round led by Versant Ventures and OrbiMed Advisors. The funds will help Iecure advance its pipeline of up to 13 programs, all of which benefit from gene editing and in vivo delivery approaches, the company said.

Iecure holds exclusive licensing rights to three liver disorder programs currently being developed in the laboratory of Dr. James Wilson, PhD, of the University of Pennsylvania Gene Therapy Program (GTP).

Under a joint research and development agreement, GTP will continue to lead preclinical research activities until Iecure files investigational new drug applications. Following the filing of its IND applications, Iecure said it will execute the clinical studies and prepare for potential commercialization of its therapies.

'Dimmable' switch for gene therapy shows potential for rare diseases
A new alternative splicing system called Xon can modulate levels of protein expression in gene therapy, much like a dimmer switch. The invention addresses...

G2 Bio Companies launch to develop genetic-based medicines
The G2 Bio Companies have launched to fund and develop gene therapy candidates from the University of Pennsylvania Gene Therapy Program.

Precision BioSciences reacquires rights to allogeneic CAR T programs
Precision BioSciences has entered into an agreement to reacquire global rights to all chimeric antigen receptor (CAR) T partnered programs covered under...

Precision BioSciences demonstrates long-term efficacy of genome editing
Precision BioSciences has demonstrated the use of its Arcus genome editing platform to achieve long-term stable reduction of low-density lipoprotein cholesterol...

Precision BioSciences nabs patent for CAR construct tech
Precision BioSciences has received a notice of allowance from the U.S. Patent and Trademark Office for patent application, No. 16/908, 030. The patent...