Congruence Therapeutics Inc. has closed a $39.5 million financing to advance into its portfolio of small-molecule correctors for diseases of protein misfolding into the clinic.
Tessera Therapeutics Inc.’s lead in vivo gene editing program, TSRA-196, has been awarded orphan drug and fast track designations by the FDA for adults with α-1 antitrypsin deficiency (AATD).
The U.S. FDA has cleared Tessera Therapeutics Inc.’s IND application for TSRA-196, its lead in vivo gene editing therapy for α-1 antitrypsin deficiency (AATD). The Australian Human Research Ethics Committee (HREC) has also granted approval for the company to begin the phase I/II clinical study.
Regeneron Pharmaceuticals Inc. and Tessera Therapeutics Inc. have established a global collaboration to develop and commercialize TSRA-196, Tessera’s lead investigational in vivo gene editing program for the treatment of α-1 antitrypsin deficiency (AATD).
Korro Bio Inc.’s latest update on RNA editing prospect KRRO-110 may mean one less competitor in alpha-1 antitrypsin deficiency (AATD), and shares of the firm (NASDAQ:KRRO) closed Nov. 13 at $6.50, down $24.92, or 79%. As part of the third-quarter earnings report, Korro said KRRO-110 produced functional protein in AATD patients but fell short of projected levels of functional protein after a single administration.
Alpha-1 antitrypsin deficiency (AATD) is a monogenic disease caused by mutations in the SERPINA1 gene, which encodes alpha-1 antitrypsin (AAT), a serine protease inhibitor mainly produced by hepatocytes.
Congruence Therapeutics Inc. has closed a $32 million financing round to advance its pipeline of small-molecule correctors for diseases of protein misfolding.
As Wave Life Sciences Ltd. released more results from its ongoing phase Ib/IIa study of small interfering RNA editing oligonucleotide WVE-006 for treating alpha-1 antitrypsin deficiency, the company’s stock (NASDAQ:WVE) dropped by 16.8% to close at $8 on Sept. 3.
As it prepares to advance its lead RNA editing candidate, AIR-001, into a phase I/II trial for alpha-1 antitrypsin deficiency, Airna Corp. Inc. closed an oversubscribed $155 million series B financing less than a year after completing its series A round. The company, based in Cambridge, Mass., with research operations in Tübingen, Germany, focuses not only on repairing harmful genetic variants found in rare genetic disorders, but also on introducing beneficial variants that improve health in common conditions.
