Immusoft of CA Inc., a wholly owned subsidiary of Immusoft Corp., has announced that the FDA has granted orphan drug designation to ISP-002, the company’s investigational engineered B-cell therapy for the treatment of mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome.

Fat tissue balances energy by storing lipids during times of abundance and mobilizing them when needed, yet sustained metabolic stress demands mechanisms that limit excessive lipid loss. Researchers at the University of California San Diego (UCSD) report that stress-induced fat breakdown triggers a neutrophil response in visceral adipose tissue that feeds back to restrain lipolysis and preserve lipid reserves.

Gene editing can repair mutations that prematurely halt protein synthesis, resulting in incomplete peptides that cause various diseases. However, other approaches achieve the same effect without altering the genome. Startup Alltrna Inc. has developed a strategy based on transfer RNA (tRNA) to bypass the premature stop codons that end early protein translation. The company already has a first clinical candidate that could treat metabolic diseases such as methylmalonemia (MMA) or phenylketonuria (PKU).

Zealand Pharma A/S and OTR Therapeutics (Shanghai) Co. Ltd. have signed a multi-program strategic collaboration and license agreement to discover and develop novel therapeutics for metabolic diseases.

Phenylketonuria (PKU) is an autosomal recessive disorder that results in decreased metabolism of the amino acid phenylalanine. Untreated PKU can lead to intellectual disability, seizures, behavioral problems and mental disorders. This metabolic disease is caused by mutations in the phenylalanine hydroxylase (PAH) gene, resulting in patients’ inability to convert phenylalanine.

CSPC Pharmaceutical Group Ltd. has obtained IND clearance from the FDA to conduct clinical trials in the U.S. with SYH-2069 for weight management in individuals with obesity or overweight and at least one weight-related comorbidity.

Neurimmune AG has expanded its transthyretin amyloid cardiomyopathy (ATTR-CM) collaboration with Alexion, Astrazeneca Rare Disease by entering into an exclusive global collaboration and license agreement to develop NI-009, a fibril depleter for light chain (AL) amyloidosis.

Junevity Inc. has raised $10 million in new funding, bringing its seed financing to a total of $20 million. The new funding will enable Junevity to advance JUN-01, its lead siRNA program for type 2 diabetes and obesity, through IND-enabling and initial clinical studies.