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.

Rznomics Inc. has received clinical trial notification (CTN) from Australia’s Therapeutic Goods Administration (TGA) for the initiation of a phase I/IIa trial evaluating RZ-004, a gene therapeutic candidate for autosomal dominant retinitis pigmentosa with rhodopsin mutation.

Epigenetic desilence of the paternal allele of the gene that causes Angelman syndrome (AS) could be used to treat this disease for which there are currently no approved therapies.

Patients with congenital hearing loss could benefit from a gene therapy currently in development. Although there are approaches that could reverse the process in children and young people before it becomes severe, so far, adults do not have any treatment that prevents the progressive deterioration of auditory sensory cells caused by this disease.

Patients with congenital hearing loss could benefit from a gene therapy currently in development. Although there are approaches that could reverse the process in children and young people before it becomes severe, so far, adults do not have any treatment that prevents the progressive deterioration of auditory sensory cells caused by this disease.