Gray platelet syndrome is an autosomal recessive platelet disorder characterized by macrothrombocytopenia and deficiency or decreased levels of alpha granules that confer a grayish appearance to the platelets. The genetic cause is located at chromosome locus 3p21, affecting the NBEAL2 gene.
Previous research has suggested that factor VIII (FVIII) can regulate the osteoprotegerin (OPG)/RANKL system, which appears to play a role in hemophilic arthropathy. Investigators have now aimed to measure the OPG levels in patients with hemophilia A/B and assess their correlation with the levels of FVIII/FIX.
Glanzmann thrombasthenia (GT) is a rare bleeding disorder caused by defects in the expression of platelet surface integrins, such as integrin alpha-IIb (GPIIb, encoded by ITGA2B).
The Biotechnology Innovation Organization (BIO) found in a new study that 77% of clinical programs focused on pain therapeutics five years ago are no longer active and that financings of companies working in the space are lackluster at best. Meanwhile, oncology companies, targeting an overall smaller market, have raised huge sums of venture capital money, $9.7 billion in 2021 vs. pain and addiction companies’ $228 million.
With the passage of the Inflation Reduction Act in the U.S. in August 2022, biopharma company leaders have re-evaluated pipelines, sought legal advice, and discussed ways to mitigate the potential impacts the legislation will have on pricing therapies and extending their reach to new indications.
The market downturn has left many biopharma companies searching for new ways to raise funds, with some eyeing the strong venture capital market as a potential resource. But having a disruptive technology and solid data may be the best way to stand out in a sea of companies, say financial executives that participated Feb. 6 in a panel discussion during the first full day of the BIO CEO 2023 conference in New York.
GSR Ventures, a venture firm focused on early stage digital health companies, forecasts a much brighter investment picture for 2023 following a hard reset in 2022, said GSR Partner Sunny Kumar in an interview with BioWorld at the J.P. Morgan Healthcare Conference in San Francisco.
Outcomes after therapy with DNA-damaging agents such as irinotecan or oxaliplatin have been seen to improve in patients with homologous recombination-deficient colorectal cancer (HRD CRC) compared to those who have HR proficient CRC (HRP CRC).
Genetically modified derivatives of the chimeric oncolytic virus (OV) CF33 have previously shown cancer selectivity and oncolytic potency against various solid tumors.
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