Search Results for “EC” – Page 304 – 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
- 106
- 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
- 201
- 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
- 122
- 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
- 146
- 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
- 177
- 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
- 98
- January 18, 2024
Simple DNA test Capable of Identifying 18 Early-Stage Cancers Could Be a Game Changer

Cancer, responsible for one-sixth of all global deaths, presents a formidable challenge, particularly because early detection is crucial for improving survival rates. However, current screening tests often fall short due to factors like invasiveness, cost, and limited accuracy in detecting early-stage diseases. In response to this challenge, researchers have now developed an innovative blood test that can identify 18 types of early-stage cancers across various major organs in the human body. This groundbreaking test from Novelna Inc. (Palo Alto, CA, USA) leverages a gender-specific panel of 10 proteins, providing a significant advancement in the ongoing battle against cancer. The foundation for the test was laid by pioneering research that underscores the potential for a new class of cancer screening tests, promising enhanced accuracy, reduced cost, and a move towards personalized healthcare. In their research, the Novelna team analyzed plasma samples from 440 individuals, encompassing both healthy subjects and those diagnosed ...
- Source: drugdu
- 233
- January 17, 2024
FDA Clears AnX Robotica’s NaviCam SB for Expanded Indications

Mike Hollan The device can produce AI-assisted readings. FDA announced that it has cleared the NaviCam Small Bowel Video Capsule Endoscopy for expanded indications. In a press release, the device’s producer AnX Robotica, revealed the agency’s decision. According to the company, the NaviCam SB is one of the most advanced pieces of technology available for small bowel video capsule endoscopy. The device uses AI to assist medical professionals as they perform tests. FDA also cleared the company’s NaviCam Tether to be used as an accessory with the NaviCam SB. These devices are designed to work together and can improve the visualization process of the esophagus. In a press release, AnX Robotica’s vice president of marketing and product management Stu Wildhorn said, “With FDA clearance, NaviCam Small Bowel Capsule Endoscopy represents a groundbreaking leap forward, now extending its revolutionary diagnostic capabilities to patients as young as 2 years old. Furthermore, clinicians ...
- Source: drugdu
- 145
- January 17, 2024
Drug Farm candidate gains rare paediatric disease status

Drug Farm has been granted a rare paediatric disease (RPD) designation from the US Food and Drug Administration (FDA) for DF-003 to treat retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis and headache (ROSAH) syndrome. DF-003 is an alpha-kinase 1 (ALPK1) indicated for the treatment of the rare autosomal dominant disorder ROSAH syndrome. The condition is caused by mutations in the ALPK1 gene, characterised by the symptoms listed in its name. ROSAH syndrome currently has no approved treatments. Drug Farm received FDA clearance of its investigational new drug (IND) application to initiate Phase I studies for the candidate in June 2023, after pre-clinical studies in ROSAH syndrome transgenic mouse models showed significant activity and favourable drug-like properties. If a new drug application for DF-003 is approved by the FDA, the company may be eligible to receive a priority review voucher (PRV) that can be redeemed to receive for any later marketing ...
- Source: drugdu
- 146
- January 17, 2024
Announcement of Zhejiang Hai Zheng Pharmaceutical Co., Ltd. on the Passing of Consistency Evaluation of Generic Drugs for Cycloserine Capsules of the Company

Zhejiang Hai Zheng Pharmaceutical Co., Ltd. (hereinafter referred to as the “Company” or “Haizheng Pharmaceuticals”) has received a Notice of Approval of Supplementary Application for Cycloserine Capsule from the State Drug Administration (hereinafter referred to as the “State Drug Administration”), and the Company’s drug Cycloserine Capsule has passed the generic drug consistency evaluation. (hereinafter referred to as “the State Drug Administration”) issued the “Drug Supplement Application Approval Notice” for Cycloserine Capsule, and the Company’s drug Cycloserine Capsule has passed the consistency evaluation of the quality and efficacy of generic drugs. Cycloserine Capsule is suitable for the treatment of active pulmonary tuberculosis and extrapulmonary tuberculosis (including renal tuberculosis) caused by tuberculosis bacteria that are sensitive to the drug and have been poorly treated with first-line anti-tuberculosis drugs (e.g., streptomycin, isoniazid, rifampicin and ethanol). Cycloserine capsules were developed by Eli Lilly and Company in the United States, and were launched in the ...
- Source: drugdu
- 82
- January 17, 2024

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