After delaying a June PDUFA date, the U.S. FDA has approved Kalvista Pharmaceuticals Inc.’s Ekterly (sebetralstat) for hereditary angioedema (HAE) in those aged 12 and older. The plasma kallikrein inhibitor now joins a market with previously approved drugs for the rare, genetic, life-threatening condition, as well as other companies with HAE drugs in development. Ekterly is the first orally-delivered on-demand treatment, as all others in the U.S. are intravenously or subcutaneously administered.
Wuhan Healthgen Biotechnology Co. Ltd. gained clearance from the Shanghai Stock Exchange July 1 to list under a recently reinstated IPO growth tier geared towards supporting “unprofitable” biotechnology firms.
Researchers at Cincinnati Children’s Hospital Medical Center have developed a new human cell model for VEXAS syndrome, a rare, severe disorder marked by systemic inflammation, bone marrow failure and high mortality. VEXAS (short for vacuoles, E1-enzyme, X-linked, autoinflammatory, somatic) is a recently identified, acquired clonal hematopoietic disease that often co-occurs with myelodysplastic syndrome.
Agonists of immune checkpoint modulators for treating autoimmune and inflammatory disorders have shown high potential in immunotherapy. P-selectin glycoprotein ligand 1 (PSGL-1) is known to exert immune checkpoint regulator functions in T cells aside from its classic role as an adhesion molecule involved in leukocyte trafficking.
Just a month after laying off 147 employees and announcing plans to mull “strategic alternatives,” Vor Biopharma Inc. reported raising $175 million in private placement in public equity financing and inking a new $4.23 billion license deal for Yantai Rongchang Biotechnologies (Remegen) Co. Ltd.’s telitacicept, a dual-target fusion protein drug approved in China for three autoimmune indications. The news was disclosed after U.S. market hours June 25. Vor’s shares (NASDAQ:VOR) gained 34 cents, or 60.5%, to close June 26 at 89 cents. The company’s shares had risen for eight consecutive trading days since June 17.
Just a month after laying off 147 employees and announcing plans to mull “strategic alternatives,” Vor Biopharma Inc. reported raising $175 million in private placement in public equity financing and inking a new $4.23 billion license deal for Yantai Rongchang Biotechnologies (Remegen) Co. Ltd.’s telitacicept, a dual-target fusion protein drug approved in China for three autoimmune indications. The news was disclosed after U.S. market hours June 25. Vor’s shares (NASDAQ:VOR) gained 34 cents, or 60.5%, to close June 26 at 89 cents. The company’s shares had risen for eight consecutive trading days since June 17.
In recognition of the fact that diversity, equity and inclusion are necessary prerequisites for precision medicine, the European Academy of Neurology announced the launch of a DEI Hub at its 11th Congress, which is being held in Helsinki through June 24.
Cue Biopharma Inc. has received pre-IND feedback from the FDA to discuss the proposed development plan for CUE-401, its lead autoimmune asset. Based on FDA feedback, the company intends to file an IND pending completion of final IND-enabling studies.
In recognition of the fact that diversity, equity and inclusion (DEI) are necessary prerequisites for precision medicine, the European Academy of Neurology (EAN) announced the launch of a DEI Hub at its 11th Congress, which is being held in Helsinki through June 24. “We know now that when we talk about personalized medicine, we have to understand that talking about stroke, for example, in a woman is different than talking about stroke in a man,” EAN president Elena Moro told the audience at the opening session of the conference.
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