Microdeletion syndrome

Zynerba Pharmaceuticals Reports Third Quarter 2022 Financial Results and Operational Highlights

Retrieved on: 
Monday, November 14, 2022
Marketing, Anxiety, Autism, Behavior, Patent, Security (finance), Form 10-K, Press release, Growth, INSPIRE, ASD, Royal commission, NDA, LPC, Bill, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Infection, Clinical Global Impression, Fragile X syndrome, SSBP, COVID-19, The Society, Poster, NORD, FDA, Fast Track, European Commission, Degenerative disease, National Organization for Rare Disorders, FXS, Depression, Annual report, Canaccord Genuity, Food, Skin, Microdeletion syndrome, DiGeorge syndrome, Research, Fragile, Patient, Pharmaceutical industry, Jean-François Zygel, Cannabidiol

DEVON, Pa., Nov. 14, 2022 (GLOBE NEWSWIRE) -- Zynerba Pharmaceuticals, Inc. (Nasdaq: ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for orphan neuropsychiatric disorders, today reported financial results for the third quarter ended September 30, 2022, and provided an overview of recent operational highlights and a pipeline update.

Key Points: 
  • $55.9 million in cash and cash equivalents at September 30, 2022; Cash runway into first quarter 2024
    DEVON, Pa., Nov. 14, 2022 (GLOBE NEWSWIRE) -- Zynerba Pharmaceuticals , Inc. (Nasdaq: ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for orphan neuropsychiatric disorders, today reported financial results for the third quarter ended September 30, 2022, and provided an overview of recent operational highlights and a pipeline update.
  • The Company currently plans to initiate a Phase 3 program in children and adolescents with 22q following topline results from RECONNECT.
  • Research and development expenses were $5.0 million for the third quarter of 2022, including stock-based compensation of $0.5 million.
  • General and administrative expenses were $3.5 million in the third quarter of 2022, including stock-based compensation expense of $0.6 million.

Scorpion Therapeutics to Present Preclinical Data for Potential Best-in-Class Mutant-Selective PI3Kα Inhibitor at San Antonio Breast Cancer Symposium

Retrieved on: 
Monday, November 21, 2022
Science, Biotechnology, Research, Pharmaceutical, Oncology, Health, Health Technology, Clinical Trials, Pharmacology, Breast cancer, Therapy, Scorpion, Microdeletion syndrome, Precision medicine, Cancer, Neoplasm, CNS, IND, Regulation of tobacco by the U.S. Food and Drug Administration, Central nervous system, Food, Patient, Pharmaceutical industry

Scorpion Therapeutics, Inc. (Scorpion), a pioneering oncology company redefining the frontier of precision medicine through its Precision Oncology 2.0 strategy, today announced that it will present preclinical data on STX-478, its potential best-in-class mutant-selective PI3K inhibitor, in a poster session at San Antonio Breast Cancer Symposium (SABCS) in San Antonio, Texas, taking place December 6 10, 2022.

Key Points: 
  • Scorpion Therapeutics, Inc. (Scorpion), a pioneering oncology company redefining the frontier of precision medicine through its Precision Oncology 2.0 strategy, today announced that it will present preclinical data on STX-478, its potential best-in-class mutant-selective PI3K inhibitor, in a poster session at San Antonio Breast Cancer Symposium (SABCS) in San Antonio, Texas, taking place December 6 10, 2022.
  • STX-478 is a highly selective, allosteric and CNS-penetrant inhibitor of mutant PI3K, designed to improve outcomes in patients harboring tumors with prevalent PI3K kinase or helical domain mutations.
  • Scorpion has completed IND-enabling studies for STX-478 and expects to submit an IND application to the U.S. Food and Drug Administration (FDA) in the first quarter of 2023.
  • We are excited to present preclinical data that further support STX-478s potential as a best-in-class PI3K inhibitor and advances its development as a novel therapeutic for the treatment of many solid tumors that are underserved by existing options, said Axel Hoos, M.D., Ph.D., Chief Executive Officer of Scorpion Therapeutics.

Coravant Expands Global Site Reliability Practice for Observability by Incorporating Open Source Service Level Objective Development Lifecycle (SLODLC)

Retrieved on: 
Thursday, November 17, 2022
Software, Technology, Networks, Security, Etsy, OutSystems, Service level, Virtusa, SRE, Accenture, Ford Motor Company, Shop, Microdeletion syndrome, Trusted Shops, Furo, Organization, IAG, Object (computer science), Risk management, Renewable energy, Oracle

Coravant, leader in leveraging leading-edge technologies to operationalize innovation for enterprises, today announced it is adopting Service Level Objective Development Lifecycle (SLODLC) as a core asset of their modernization practice.

Key Points: 
  • Coravant, leader in leveraging leading-edge technologies to operationalize innovation for enterprises, today announced it is adopting Service Level Objective Development Lifecycle (SLODLC) as a core asset of their modernization practice.
  • The practice will focus on introducing observability to support customers who need site reliability engineering (SRE) and application engineering professional services using SLODLC.
  • Introduced in May 2022, the SLODLC community is quickly moving to become the de facto standard for Service Level Objectives (SLOs) and their scalable deployment.
  • They are a go-to partner for complex transformations and migrations for some of the largest, most critical enterprise customers.

Biosimilar Growth Hormones Global Market Report 2022: Growing Prevalence of Growth Hormone Deficiency in Children Driving Sector - ResearchAndMarkets.com

Retrieved on: 
Wednesday, November 16, 2022
Health, General Health, Arthralgia, Turner syndrome, Skin, Idiopathic short stature, Microdeletion syndrome, RDNA, Carpal tunnel syndrome, Route of administration, DNA, Swelling, Insulin, Edema, Life, Short stature, Child, Application, Pituitary gland, Growth, Diabetes, Hypoesthesia, COVID-19, Infection, Nutrient, Risk, Distribution, HGH, Hypothalamus, Pharmaceutical industry, Birth control

The "Biosimilar Growth Hormones Global Market Report 2022: By Route, By Application, By Distribution" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Biosimilar Growth Hormones Global Market Report 2022: By Route, By Application, By Distribution" report has been added to ResearchAndMarkets.com's offering.
  • The global biosimilar growth hormones market is expected to grow from $0.59 billion in 2021 to $0.64 billion in 2022 at a compound annual growth rate (CAGR) of 8.4%.
  • The growing number of children suffering from growth hormone deficiency is driving the growth of the global biosimilar growth hormone market.
  • The growing awareness about the side effects caused by the continuous usage of the biosimilar growth hormones is expected to restrict the growth of the market.

Jennifer S. Wilkov, Producer of the October Global Speak Up Women Conference, Awards New York State the Make A Difference Award

Retrieved on: 
Friday, November 18, 2022
Sex, Trauma, Make, Human, Human spirit, Workplace, Conference, UN, Adult, Bipartisanship, Diversity, Friends, Empowerment, Survivor, CNBC, United Kingdom, Extraversion and introversion, Sexual assault, Courage, NASA, Book, Shame, Harvard Business Review, DEI, Microdeletion syndrome, Violence, Diversity Woman, Sex industry, Interior design, Woman

BROOKLYN, N.Y., Nov. 18, 2022 /PRNewswire/ -- Jennifer S. Wilkov, multi #1 international best-selling award-winning author, founder, and CEO of Speak Up World LLC, (http://www.SpeakUpWomen.com), successfully produced the 2022 October Global Speak Up Women Conference virtually online on Saturday, October 29, 2022. Speakers and participants from thirteen states and the United Kingdom, Germany, and others engaged in virtual talks about the importance of speaking up in women's personal and professional relationships and for causes she cares about to create change where change is necessary.

Key Points: 
  • "Speak Up Women is the #1 resource for women who want to speak up in their lives for the lives they imagine.
  • To learn more about the Speak Up Women Community, conferences, events, and the quarterly subscription box program, go to https://www.SpeakUpWomen.com .
  • It's a community for women to connect, collaborate, create, and celebrate each other amongst colleagues and friends.
  • It helps women connect with like-minded individuals in support of mutually aligned interests, passions, and values.

Albert Einstein College of Medicine Researchers Develop Promising New Cancer Therapy

Retrieved on: 
Tuesday, November 15, 2022
Albert Einstein College of Medicine, Technology, NK, Immunotherapy, Immunity, Student, YouTube, Microbiology, College, Melanoma, Ageing, Squamous cell carcinoma, University Hospital (Newark, New Jersey), AIDS, Diabetes, National, Lung, Attention, Lymphoma, PVR, Stomach, Partnership, Myeloid leukemia, Liver disease, Patient, Degenerative disease, Protein, Pembrolizumab, Paper, Pancreatic cancer, College of Medicine, Doctor of Philosophy, Montefiore, Microdeletion syndrome, Journal, Urology, Teaching hospital, Journal of Clinical Investigation, Multimedia, College of Medicine (UK), Head, Neuroscience, Neoplasm, JCI, Neck, TIGIT, Acute myeloid leukemia, Research, Patent, Albert Einstein, Large-cell lung carcinoma, Cancer research, Pharmaceutical industry, Vaccine, Cancer, Medicine, Facebook

Their introduction a decade ago marked a major advance in cancer therapy, but only 10% to 30% of treated patients experience long-term improvement.

Key Points: 
  • Their introduction a decade ago marked a major advance in cancer therapy, but only 10% to 30% of treated patients experience long-term improvement.
  • In a paper published online today in The Journal of Clinical Investigation (JCI), scientists at Albert Einstein College of Medicine describe findings that could bolster the effectiveness of immune-checkpoint therapy.
  • Rather than rally T cells against cancer, the Einstein research team used different human immune cells known as natural killer (NK) cellswith dramatic results.
  • About Albert Einstein College of Medicine Albert Einstein College of Medicine is one of the nation's premier centers for research, medical education and clinical investigation.

TScan Therapeutics Reports Third Quarter 2022 Financial Results and Provides Business Update

Retrieved on: 
Wednesday, November 9, 2022
Umbrella, IND, HCT, Immunotherapy, Research, Therapy, Microdeletion syndrome, MAGE-A1, Securities Exchange Act of 1934, Company, MDS, Novartis Institute for Tropical Diseases, Review, Biomedical Research, Annual general meeting, Food, Society, Hand, Society for Immunotherapy of Cancer, Collection, Cancer, PRAME, AML, Patient, Analysis, Result, ASH, COVID-19, Private Securities Litigation Reform Act, TCR, Nasdaq, Acute myeloid leukemia, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Lymphatic system, Hlas, ALL, HLA, Clinical trial, Acute leukemia, Growth, IND World's Fair Line, FDA, Trial of the century, GLOBE, Haematopoietic system, Financial condition report, HPV, Annual report, Safety, RIC, Multiplexing, SITC, Pharmaceutical industry, Vaccine, Food delivery

WALTHAM, Mass., Nov. 09, 2022 (GLOBE NEWSWIRE) -- TScan Therapeutics, Inc. (Nasdaq: TCRX), a clinical-stage biopharmaceutical company focused on the development of T cell receptor (TCR)-engineered T cell therapies (TCR-T) for the treatment of patients with cancer, today reported financial results and provided a business update for the third quarter ended September 30, 2022.

Key Points: 
  • The initial $30 million that we drew at the close provides TScan with a cash runway into the second quarter of 2024.
  • During the quarter TScan announced the closing of a convertible debt facility for up to $60 million with K2 HealthVentures (K2HV).
  • As of September 30, 2022, TScan Therapeutics had cash and cash equivalents of $137.3 million, excluding $5.0 million of restricted cash.
  • For the third quarter ended September 30, 2022, TScan Therapeutics reported a net loss of $16.2 million, compared to a net loss of $15.8 million for the 2021 Quarter.

ExCellThera announces 100th patient treated with UM171-expanded cell therapy

Retrieved on: 
Tuesday, November 8, 2022
ATMP, GLOBE, Health, Adult, Doctor of Philosophy, TRM, Rejuvenation, Cell therapy, Center for Cell and Gene Therapy, Degenerative disease, Incidence, Gene therapy, Classification, Health Canada, Type, GRFS, DMF, Microdeletion syndrome, Safety, Culture, HSC, Regenerative medicine, EMA, HLA, Patient, GMP, MD, Leukemia, FDA, Caregiver, Pharmaceutical industry

MONTREAL, Nov. 08, 2022 (GLOBE NEWSWIRE) -- ExCellThera Inc. (ExCellThera), a world leader in blood stem cell expansion and rejuvenation, announced today that its proprietary ECT-001 Cell Therapy has been administered to 100 patients.

Key Points: 
  • MONTREAL, Nov. 08, 2022 (GLOBE NEWSWIRE) -- ExCellThera Inc. (ExCellThera), a world leader in blood stem cell expansion and rejuvenation, announced today that its proprietary ECT-001 Cell Therapy has been administered to 100 patients.
  • ECT-001 Cell Therapy is an investigational UM171-expanded cord blood cell therapy being evaluated in trials across multiple adult and pediatric hematological indications, including severe leukemias and myelodysplastic syndromes, in the United States and Canada.
  • ExCellTheras lead proprietary molecule, UM171, has become the standard for hematopoietic stem cell (HSC) expansion in Cell and Gene Therapy.
  • In addition to ECT-001 Cell Therapy, ExCellThera also has a pre-clinical pipeline of next generation cell and gene therapies in adjacent fields of use.

FibroGen Announces $50 Million Royalty Monetization Financing with NovaQuest Capital Management

Retrieved on: 
Monday, November 7, 2022
Astellas Pharma, Dialysis, GLOBE, CIA, Idiopathic pulmonary fibrosis, U.S. Securities and Exchange Commission, Pancreatic cancer, LAPC, CKD, Risk, Research, Biology, Morgan Stanley, Pamrevlumab, Specialization, CTGF, SEC, NASDAQ, Microdeletion syndrome, Duchenne muscular dystrophy, Time, Company, Human, Duchenne, Patient, MDS, Growth, Therapy, IPF, DMD, Anemia, Animal, Security (finance), Agreement, Generic drug, Risk management, Car rental, Pharmaceutical industry, Roxadustat

Supported by global launches of EVRENZO in the territories partnered with Astellas Pharma Inc.

Key Points: 
  • Supported by global launches of EVRENZO in the territories partnered with Astellas Pharma Inc.
    SAN FRANCISCO, Nov. 07, 2022 (GLOBE NEWSWIRE) -- FibroGen, Inc. (NASDAQ: FGEN) today announced a royalty monetization financing with NovaQuest Capital Management (NovaQuest) that will result in proceeds to FibroGen in the amount of $50 million, bringing non-dilutive capital to drive innovation and growth.
  • The financing provides capital to expand and advance the company's wholly-owned late-stage pipeline toward commercialization.
  • Executing this deal in the current market environment speaks to the quality of the assets and the aligned confidence in the commercial opportunity of EVRENZOby NovaQuest Capital Management."
  • NovaQuest Capital Management, located in North Carolinas Research Triangle, is a life science investment firm with a specialization in biopharmaceuticals.

FibroGen Reports Third Quarter 2022 Financial Results

Retrieved on: 
Monday, November 7, 2022
Dialysis, Adult, Pamrevlumab, CKD, LAPC, Research, Biology, Sale, CTGF, Microdeletion syndrome, Company, Duchenne, MDS, Commonwealth, Security (finance), AstraZeneca, Review, Marketing, Erythropoietin, Pancreatic cancer, Food, SEC, U.S. Securities and Exchange Commission, Erythropoiesis, Chronic kidney disease, Patient, DMD, Scar, Organ dysfunction, Webcast, JDE, Mission statement, Duchenne muscular dystrophy, NDD, Anemia, Income, SAN, GLOBE, CIA, Idiopathic pulmonary fibrosis, Investment, Risk, Orphan, Annual report, Failure, NASDAQ, Fast Track, Safety, ODD, IPF, Therapy, Video game, Pharmaceutical industry, Music, Medical device, Medicine, Food delivery, Animal, Cryptocurrency, Bookkeeping, Astellas Pharma, Roxadustat, EU

SAN FRANCISCO, Nov. 07, 2022 (GLOBE NEWSWIRE) -- FibroGen, Inc. (NASDAQ: FGEN) today reported financial results for the third quarter 2022 and provided an update on the companys recent developments.

Key Points: 
  • SAN FRANCISCO, Nov. 07, 2022 (GLOBE NEWSWIRE) -- FibroGen, Inc. (NASDAQ: FGEN) today reported financial results for the third quarter 2022 and provided an update on the companys recent developments.
  • Third quarter total roxadustat net sales in China1 by FibroGen and the distribution entity (JDE) jointly owned by FibroGen, and AstraZeneca was $59.0 million, compared to $57.8 million in the third quarter of 2021.
  • At September 30, 2022, FibroGen had $441.6 million in cash - defined as cash, cash equivalents, investments, and accounts receivable.
  • FibroGen will host a conference call and webcast today, Monday, November 7, 2022, at 5:00 p.m. Eastern Time (2:00 p.m. Pacific Time) to discuss financial results and provide a business update.