Ending a late 2021 deal that was potentially worth billions, Moderna Inc. and Metagenomi Inc. are going their separate ways. The two had been collaborating on gene-editing R&D to develop therapies for treating serious genetic diseases. Moderna said it agreed with Metagenomi to end the deal as “Moderna continues to strategically prioritize its research and development investments.”
Palo Alto, Calif.-based Bridgebio Pharma Inc. will hand over development and sales of its rare bone growth disorder therapy, infigratinib, in Japan to Kyowa Kirin Co. Ltd. under its latest exclusive licensing deal.
Palo Alto, Calif.-based Bridgebio Pharma Inc. will hand over development and sales of its rare bone growth disorder therapy, infigratinib, in Japan to Kyowa Kirin Co. Ltd. under its latest exclusive licensing deal.
Reneo Pharmaceuticals Inc. is halting development of its only asset, mavodelpar, after a phase IIb study missed its primary and secondary efficacy endpoints. The selective peroxisome proliferator-activated receptor (PPAR)-delta agonist was being developed to treat to rare genetic mitochondrial diseases.
In its bid to become, as Chairman and CEO David Hallal said, the “world’s most indispensable cell and gene therapy technology company,” Elevatebio LLC disclosed a $401 million series D round with support from new and existing investors. At the same time, the company’s Life Edit Therapeutics Inc. affiliate inked a potential billion-dollar collaboration focused on gene editing therapies.
Chimeric antigen receptor T-cell therapies and mRNA-based vaccines represent two of the most significant new modalities to gain regulatory approval in the past decade. Capstan Therapeutics Inc. has emerged from stealth with bold ambitions to combine these two approaches in mRNA-programmed cell therapies that will be generated in vivo from patients’ endogenous cells. It has so far secured $165 million in equity funding to pursue that vision.
Orna Therapeutics Inc., which is pioneering a novel circular RNA protein expression technology in several therapeutic areas, has achieved lift-off. The Cambridge, Mass.-based company has closed a broadly based alliance in infectious disease and oncology with Merck & Co. Inc., under which it is getting $150 million up front and up to $3.5 billion in development, regulatory and sales-based milestones. In addition, Merck, of Rahway, N.J., is investing another $100 million in Orna’s equity, as part of its $221 million series B round, which the company also disclosed on Aug. 16.
Fabric Genomics Inc.’s Gem artificial intelligence algorithm plus whole genome and whole exome data detected more than 90% of disease-causing variants in infants with rare diseases, a study in Genome Medicine demonstrated. The full process from blood sample to shortlist of causative variants and likely diseases takes just a matter of hours and the time to interpret whole genomes is condensed to about 15 minutes.
Metagenomi Inc. has raised a $65 million series A financing to expand its gene editing abilities, advance its research and validate its pipeline in preclinical studies. The company’s CRISPR-based systems use algorithms for screening thousands of genomes from microorganisms to advance therapies for use in oncology, genetic diseases and possibly much more.