Carmine Therapeutics Inc. said Tuesday that Takeda Pharmaceutical Co. Ltd. has engaged it in a research collaboration aimed at the discovery, development and commercialization of new nonviral gene therapies for two rare disease targets. Terms of the deal included an up-front payment of undisclosed value for Carmine, plus research funding, and more than $900 million in potential milestone payments. Read More
DUBLIN – Gene therapy developer Freeline Therapeutics Ltd. added $80 million in new investment to take its series C round to $120 million in total. The new cash will help to fund a pivotal trial of its lead gene therapy program in hemophilia B, enable it to continue a phase I/II trial of a gene therapy in Fabry disease and allow it to progress its preclinical programs in Gaucher disease and hemophilia A, while also making ongoing investments in its adeno-associated virus (AAV) gene therapy technology and its manufacturing platform. Read More
The share prices of blue-chip biopharmaceutical companies closed out the month on a high note to contribute to their stellar collective performance during the second quarter, with the BioWorld Biopharmaceutical index increasing almost 20%. Read More
Zynerba Pharmaceuticals Inc.’s stock was slashed nearly in half June 30 as the company’s pivotal Connect-FX study for treating fragile X syndrome behavioral symptoms failed to achieve statistical significance in its primary and three key secondary endpoints. Read More
BEIJING – Cross-border biotech Denovo Biopharma LLC, based in San Diego, the U.S. and Hangzhou, China, closed a series C funding round on June 29 to pocket ¥590 million (US$83.5 million). Read More
Two separate groups have recently shown that in mouse models, inactivation of a single gene was enough to directly convert other cell types in the brain into neurons. Read More
Citing the First Amendment, the Fifth Amendment and the logic of fair play, Pfizer Inc. is taking on a Health and Human Services’ (HHS) antitrust policy that prohibits drug companies from helping Medicare beneficiaries with hefty copays. Read More
The push for a vaccine for the COVID-19 pandemic may have no parallel in pharmaceutical history, and FDA commissioner Stephen Hahn vowed in a Senate hearing that the agency will rely on the agency’s traditional standards for scientific evidence in premarket reviews of those vaccines. Read More
A month ahead of its PDUFA date, Ultragenyx Pharmaceutical Inc.’s NDA for triheptanoin (UX-007) won FDA approval. Branded Dojolvi, the highly purified, synthetic, seven-carbon fatty acid triglyceride was cleared for use in treating pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD), a group of rare and life-threatening genetic disorders in which the body is unable to convert long-chain fatty acids into energy. Read More
Biopharma happenings, including deals and partnerships, grants, preclinical data and other news in brief: Advanced Accelerator, Audentes, Basilea, Bioasis, Blueprint, Burning Rock, Camino, CNS, Cstone, Genespire, Hoth, Immatics, Marker, Meissa, Novartis, Optinose, Vertex, Ziphius. Read More
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Alterity, Ardelyx, Astrazeneca, Bioinvent, Mediwound, Merck, Nuvox, Oncopeptides, Oncternal, Osmotica, Prestige, Sparingvision, Synthetic Biologics, Vericel, Verrica. Read More
