Kano Therapeutics Inc. announced it has secured $5 million in seed funding, bringing its total funding to date to $7.1 million. The company plans to use the funding to begin internal pipeline development of ex vivo genetic medicines based on kilobase gene insertion, expand its existing therapeutic collaborations to initiate externally driven preclinical programs, and scale its production capacity.

Actio Biosciences Inc.’s ABS-0871 has received both orphan drug designation and rare pediatric disease designation from the FDA. ABS-0871 is in preclinical development for the treatment of Charcot-Marie-Tooth disease subtype 2C (CMT2C).

A spontaneous homozygous deletion affecting the copy number of the cadherin 23 (Cdh23) gene in a line of laboratory mice gave rise to a new murine model with hereditary hearing loss and vestibular alterations.

Release Therapeutics SA has announced that it has secured CHF3.3 million (US$3.87 million) in seed funding. The proceeds will be used to finance primate studies of the company’s cell macroencapsulation technology for use in metachromatic leukodystrophy (MLD).

Meta Pharmaceuticals Inc. announced that the FDA has granted rare pediatric disease designation to its investigational new drug META-001-PH for the treatment of primary hyperoxaluria (PH), an autosomal recessive metabolic disorder in which oxalate is overproduced and deposited in the body.

Airna Corp. Inc., of Cambridge, Mass., announced it has closed an oversubscribed $60 million financing round, bringing its total series A funding to $90 million.

Innorna Co. Ltd.’s IN-016 has received orphan drug status from the U.S. FDA for the treatment of progressive familial intrahepatic cholestasis (PFIC). Earlier in July, IN-016 had been granted Rare Pediatric Disease Designation. PFIC is a group of rare genetic disorders associated with defects in bile acid secretion or transport, resulting in unwanted bile accumulation within the liver.

Intellia Therapeutics Inc. has received clearance from the U.K. Medicine and Healthcare products Regulatory Agency (MHRA) to initiate a phase I/II study of NTLA-3001 for the treatment of α1-antitrypsin deficiency (AATD)-associated lung disease.

The scene has been set for research into gene misexpression across different tissues, to understand the part it plays in a range of diseases and search for new drug targets, following publication of the first comprehensive survey of where genes are active when they are expected to be switched off.

Arrhythmogenic cardiomyopathy (ACM) is a severe genetic cardiac disorder caused by mutations in some desmosomal genes. The most frequently affected gene in patients with ACM is PKP2, the loss of which provokes desmosomal instability that leads to activation of downstream disease processes ultimately resulting in life-threatening ventricular arrhythmia and heart failure.