PharmaFocus Today – Page 212 – media

Blood Test to Track Gene Expression in the Brain Could Help Prevent Neurological Diseases

Encased within a complex network of specialized blood vessels, the brain remains the body’s most protected organ. This intricate structure, while protective against external threats, poses significant challenges for researchers seeking to understand gene expression dynamics and their link to diseases. Addressing this challenge, scientists have now introduced a noninvasive method to track gene expression in the brain, potentially transforming research in brain development, cognitive function, and neurological disorders. The team of scientists at Rice University (Houston, TX, USA) has developed a groundbreaking class of molecules named released markers of activity (RMAs). These RMAs offer a noninvasive solution to measure gene expression in the brain via a simple blood test. Traditionally, assessing gene expression in the brain has been limited to post-mortem analysis or less sensitive and specific modern neuroimaging techniques. The RMA platform, however, introduces a synthetic gene expression reporter into the brain, which synthesizes a protein capable of ...
- Source: drugdu
- 121
- January 18, 2024
Four-Biomarker Panel Could Enable Early Ovarian Cancer Detection

Ovarian cancer remains a significant cause of mortality globally. While advancements in cytoreductive surgery and chemotherapy have improved survival for those with epithelial ovarian cancer, prognosis heavily depends on the stage at diagnosis. Early detection, particularly at stage I, offers a five-year survival rate exceeding 90%. However, this rate drops to around 70% for stage II, where cancer is confined to the pelvis, and declines further for later stages, plummeting to a mere 20% at stage IV. Computational models suggest that detecting ovarian cancer in stages I or II could increase the cure rate by 10-30%. Currently, Cancer Antigen 125 (CA125), a protein found in various cells, including those of ovarian cancer, is the only tumor marker recommended for clinical use in diagnosing and managing ovarian cancer. Now, a new study has shown that a panel of four biomarkers is more sensitive than CA125 alone for early ovarian cancer detection, ...
- Source: drugdu
- 194
- January 18, 2024
Vertex’s CRISPR Gene Therapy Lands Another FDA Nod in a Rare Blood Disease

The FDA approved Vertex Pharmaceuticals’ gene therapy Casgevy for treating beta thalassemia, an inherited blood disease that leads to low levels of functioning hemoglobin. Last month, the one-time treatment won its first FDA nod for treating sickle cell disease. By FRANK VINLUAN A one-time CRISPR-based gene therapy developed by Vertex Pharmaceuticals and CRISPR Therapeutics now has an additional FDA approval as a treatment for the rare blood disease beta thalassemia. The affirmative decision announced Tuesday for the therapy, Casgevy, was widely expected following its December approval in sickle cell disease, but it comes more than two months early. The target date for an FDA decision in the beta thalassemia indication was March 30. Casgevy won its first ever regulatory approval in the United Kingdom, a November decision that covered both sickle cell disease and beta thalassemia. Beta thalassemia is an inherited disease in which genetic mutations lead to low levels ...
- Source: drugdu
- 246
- January 18, 2024
Allakos Skin Drug Fails 2 Mid-Stage Tests, Sparks Restructuring to Cut 50% of Staff

Allakos’s lirentelimab did not beat a placebo in separate Phase 2 tests in atopic dermatitis and spontaneous chronic urticaria. A restructuring now turns the biotech’s focus to an early-stage drug candidate also designed to treat inflammation. By FRANK VINLUAN The lead therapeutic candidate of Allakos has failed in two mid-stage clinical trials for inflammatory skin disorders, leading the biotech to halt work on the drug. Allakos is shifting its focus to an earlier-stage program in its pipeline but will do so with fewer staff. A corporate shakeup is shaving about half of the company’s workforce. San Carlos, California-based Allakos aims to treat allergic, inflammatory, and proliferative diseases with antibodies that target receptors on cells that create immune responses in the body. By activating inhibitory receptors, these drugs are intended to stop inflammation. Its most advanced drug candidate was lirentelimab, which was designed to target an inhibitory receptor on mast cells ...
- Source: drugdu
- 146
- January 18, 2024
Roche exec says more pull incentives are needed to tackle AMR

“Pull incentives such as high cost of therapy and longer exclusivity periods, similar to those granted for orphan drugs, are needed to promote the development of new antimicrobials” says Severin Schwan, chairman of the board of directors of Roche. Schwan was speaking about the threat of antimicrobial resistance (AMR) at the World Economic Forum Annual Meeting taking place from 15–19 January 2024 in Davos, Switzerland. Shyam Bishen, head of the Centre for Health and Healthcare at the World Economic Forum, noted at the same discussion that AMR is the third leading cause of death globally and is forecasted to be responsible for 10 million deaths annually in 2050. Schwan said most companies have left the antimicrobial sector as they perceive that there is no sustainable business model. “The research and development subsidiaries offered by various governments to develop antimicrobials are a waste of money and are not worth it to ...
- Source: drugdu
- 174
- January 18, 2024
Shanghai Serum Bio-technology Co., Ltd. voluntarily discloses the announcement of obtaining the ethical review approval for Phase II clinical trial of Anti-Viper Venom Serum

Shanghai Serum Bio-technology Co., Ltd. (hereinafter referred to as the “Company”) has passed the approval of the Medical Ethics Committee of the First Affiliated Hospital of Guangzhou Medical University, the lead organization of the Phase II Clinical Study of Anti-Viper Venom Serum Injection, and obtained the ethical review approval document. The relevant information is announced as follows: The results of the completed Phase I clinical trial study of Anti-Viper Venom Serum Injection (Registration No. CRT20202621) showed that good safety and tolerability were observed in healthy subjects given different dose groups of Anti-Viper Venom Serum. The results of drug dose, drug concentration, and correlation analysis with effect indicated that the efficacy of the anti-viper venom serum (ability to neutralize viper venom in vitro) was well correlated and dependent on the dose of the test drug administered, and the concentration of the drug in the blood. Based on the positive safety results ...
- Source: drugdu
- 187
- January 18, 2024
Announcement of Changchun Hi-Tech Industrial (Group) Co., Ltd. on the Acquisition of FDA Implied License for Clinical Trials of Drugs by its Controlling Subsidiary

According to the relevant rules of the U.S. Food and Drug Administration (hereinafter referred to as “FDA”), Changchun Hi-Tech Industrial (Group) Co. (hereinafter referred to as “GenSci”) will carry out Phase I clinical trials in the United States for GenSci125 for injection (IND No. 168162), and the relevant information is hereby announced as follows: The Company had issued the ”Announcement on the Approval of the Application for Clinical Trial of GenSci125 for Injection by the Controlling Subsidiary” (Announcement No. 2023-096) on December 15, 2023, obtaining the ”Notification of Approval for Clinical Trial of Drugs” by the State Drug Administration of the PRC in respect of the project of GenSci125 for injection and agreeing to carry out the clinical trial. On December 15, 2023, GenSci obtained the IND confirmation letter from FDA. According to the requirements of the letter, there is an implied license period of 30 days after the receipt ...
- Source: drugdu
- 196
- January 18, 2024
Takeda picks up 2nd FDA-approved indication for immune globulin treatment HyQvia

It was a decade and four companies ago that HyQvia was first approved by the FDA for primary immunodeficiency (PI). Ten years later, Takeda’s subcutaneous immune globulin treatment has scored its second indication, as the FDA has approved it as a maintenance therapy for chronic inflammatory demyelinating polyneuropathy (CIDP). A progressive neurological disorder, CIDP causes weakness and reduced feeling in the arms and legs. In 2014, when it was owned by Baxter, HyQvia was endorsed to treat adults with PI, an umbrella term referring to 400-plus rare diseases that can affect the immune system. Then in April of last year, Takeda won a label expansion to treat children ages 2 to 16. The approval for CIDP was based on a phase 3 trial that enrolled 122 adults. The study showed that 14% of those on HyQvia relapsed, compared with 32% of those on placebo. Another phase 3 trial assessed the ...
- Source: drugdu
- 182
- January 18, 2024
Gilead calls off Oceanside manufacturing expansion, uproots biologics development team

Gilead Sciences is halting the expansion of its biologics site in Oceanside, California, and is moving the existing team’s responsibilities to its Foster City campus in the same state, a spokesperson confirmed. The move is based on Gilead’s desire to bring members of the biologics development and manufacturing team closer to the company’s R&D unit, which is based in the Bay Area in Foster City, the spokesperson said. “Over the next few years, the majority of our Gilead Biologics teams in our Pharmaceutical Development & Manufacturing organization, most of whom are currently in Oceanside, CA, will transition to Foster City,” the spokesperson said. The move will begin this year and conclude by 2027 “at the latest,” according to Gilead, with business operations continuing “as usual” for the time being. Oceanside and Foster City are about an 8-hour drive apart. The company did not specify how many employees, if any, would ...
- Source: drugdu
- 238
- January 18, 2024
Vertex, CRISPR’s gene-editing therapy Casgevy wins early FDA nod to treat beta thalassemia

Vertex Pharmaceuticals and CRISPR Therapeutics have scored an FDA approval for their gene-editing therapy Casgevy (exa-cel) to treat transfusion-dependent beta thalassemia (TDT). The approval comes less than six weeks after the U.S. regulator signed off on Casgevy to treat patients with sickle cell disease (SCD). With the TDT nod, Casgevy becomes the first treatment for the rare blood disorder using CRISPR gene-editing technology. Bluebird Bio also gained approval in 2022 for its gene therapy, Zynteglo, to treat TDT. The endorsement arrived more than two months early as an FDA decision was due on March 30. “On the heels of the historic FDA approval of Casgevy for sickle cell disease, it is exciting to now secure approval for TDT well ahead of the PDUFA date,” Reshma Kewalramani, M.D., Vertex’s CEO said in a release. The companies have opened nine authorized treatment centers (ATCs) to administer Casgevy to patients, Vertex said. Each ...
- Source: drugdu
- 115
- January 18, 2024

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