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Almost every one of us will know someone with a neurodegenerative disease such as Alzheimer’s or Parkinson’s. It is less common to know someone with a rare genetic disease; but while individually rare, collectively they have an enormous impact. This will, in turn, slow the progression of, or even halt, the disease.
Most rare diseases are caused by a single gene defect, but severity can vary considerably among patients. Modifier genes can help explain that variability and can alter or even prevent disease onset and progression, making them appealing therapeutic targets. However, the identification of these genes is challenging.
UBX1325 is developed from BM-962, a Bcl-xL inhibiting compound licensed to UNITY by Ascentage Pharma for the treatment of age-related diseases. This progress in clinicaldevelopment qualifies Ascentage Pharma for a milestone payment according to the terms of the licensing agreement. SUZHOU, China and ROCKVILLE, Md. ,
Human derived induced pluripotent stem cells (hiPSCs) have revolutionised research and are increasingly used for toxicology screening and disease modelling. 1-4 This approach saves time, money, and resources, and ultimately leads to safer medicines going forward to the clinic. 2018 Mar;17(3):167–81. 2018 Dec 11;94(10):390–411.
Rare Disease Day on February 28th is a chance for rare disease communities to come together as one and join in conversation with stakeholders searching for treatments, raising money to support research, and changing the way healthcare and governments interact with patients. In my case, my daughter Iris has GM1 gangliosidosis.
Making a little tangle of DNA visible to the eye and understanding that this is the basis of complex organisms, which might be altered in disease, was the defining moment for my future path in life sciences. Everything started in school with an experiment on isolating DNA from bananas. in North America) in late 2017.
2] Pre-clinical studies have shown that these differentiated properties of HMBD-001 lead to robust and sustained tumor growth inhibition in multiple HER3 cancer models, including those with NRG1-fusions. Our highly experienced teams in Singapore and the US span antibody discovery, pharmacology, production and clinicaldevelopment.
He continues: “With the green light granted by the FDA to continue our clinical research, we are one step closer to offering psoriasis patients a novel, improved oral treatment option and thereby enhancing their quality of life. 2018)2: Menter MA, Armstrong AW, Gordon KB, Wu JJ. 2018 Feb;37(2S):S48-S51.
These findings were mirrored in the clinical results from an analysis of two metastatic melanoma patient cohorts who received anti-PD-1 treatment: patients with responses and disease control had significantly lower GDF-15 serum levels (measured before treatment start), while none of the patients with elevated GDF-15 levels showed a lasting response.
Sanofi Pasteur, the vaccines global business unit of Sanofi, and Translate Bio (NASDAQ: TBIO), a clinical-stage messenger RNA (mRNA) therapeutics company, today announced the start of the Phase 1/2 clinical trial for MRT5500, an mRNA vaccine candidate against SARS-CoV-2, the virus that causes COVID-19.
Patients with high numbers of clonal neoantigens show improved disease-free survival. Sergio co-led the development of a first-in-class Treg-depleting anti-human CD25 antibody with TUSK Therapeutics, which was acquired by Roche in 2018 and is currently under clinical evaluation.
P otential to be a safer and effective alternative to the intravenous immunotherapies currently used for Crohn’s Disease.
Crohn’s Disease Therapeutics Market Size $4.7
Phase1b/2 clinical study to be conducted in the United States and several European countries. Billion by 2025.
and Europe.
Bayer is focused on addressing the various medical needs of cancer patients, providing treatments that improve patient outcomes throughout the different stages of the disease,” said Robert LaCaze, Member of the Executive Committee of the Pharmaceuticals Division and Head of the Oncology Strategic Business Unit at Bayer.
. “This marks our second strategic agreement with Asieris, a well-run and well-capitalized company that is executing ahead of plan on the clinicaldevelopment of Cevira ®, which we licensed to them in July 2019. Asieris strives to become a leading pharmaceutical company focusing on genitourinary tumors and related diseases.
333-228656) previously filed with the Securities and Exchange Commission (the “SEC”) on December 3, 2018, and declared effective by the SEC on December 26, 2018. If adequate funds are not available on a timely basis, Allena may be required to delay, limit, reduce or terminate its clinicaldevelopment of reloxaliase.
The mechanism is currently being evaluated for the potential treatment of cognitive impairment and other symptoms associated with a range of neuropsychiatric and neurodegenerative disorders, such as Alzheimer’s disease and major depressive disorder. senior vice president, R&D, chief scientific officer, AbbVie.
Growing into a biopharmaceutical company, Innovent has built an integrated platform covering from drug discovery, CMC, clinicaldevelopment and commercialization. On October 31, 2018 , Innovent was listed on the Main Board of the Stock Exchange of Hong Kong Limited with the stock code: 01801.HK.
Based on the positive Phase 1 results reported and the unprecedented need for COVID-19 vaccines, Clover and its partners are confident to enter late-stage clinicaldevelopment for both adjuvanted vaccines. The Phase 1 clinical trial was funded by the Coalition for Epidemic Preparedness Innovations (CEPI).
About non-Hodgkin’s lymphoma Non-Hodgkin’s Lymphoma (NHL) comprises a highly heterogeneous group of chronic diseases with poor prognosis. NHL is the most common hematologic malignancy and the tenth most common cancer worldwide, with about 510,000 new cases diagnosed in 2018. It accounts for about 250,000 deaths per year worldwide.
Coordinated efforts are needed to combat this disease and improve the clinical status of persons with HBV infection. Currently there is no cure for hepatitis B, however there are several drugs in clinicaldevelopment to improve the lives and clinical outcomes for persons with chronic HBV infection, including functional cure strategies.
Knopp Biosciences is a privately held drug discovery and development company focused on delivering breakthrough treatments for immunological and neurological diseases of high unmet need. Knopp’s clinical-stage oral small molecule, dexpramipexole, is in Phase 2 clinical trials in moderate-to-severe eosinophilic asthma.
(NYSE: PFE) today announced a global collaboration to develop and commercialize ARV-471, an investigational oral PROTAC® (PROteolysis TArgeting Chimera) estrogen receptor protein degrader. The estrogen receptor is a well-known disease driver in most breast cancers. Terms of the Collaboration. and other customary closing conditions.
Roche leads the clinicaldevelopment of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics. Evrysdi was granted PRIME designation by the European Medicines Agency (EMA) in 2018 and Orphan Drug Designation by the U.S. Enrolment for Part 2 was completed in November 2018.
Sairiyo is a biotechnology company focused on repurposing and developing improved formulations of naturally derived compounds for serious, rare, and life-threatening diseases with the aim to obtain European Medicines Evaluation Agency and U.S. Research and development efforts will be led by Dr. Paul Van Slyke.
The NCT03763149 study is a phase 1a clinical study evaluating the tolerability, safety, and PK/PD properties of IBI188 (letaplimab) as monotherapy for advanced malignancies that failed in standard treatments. .
The study preset 7 dose groups ranging from 0.1mg/kg to 30mg/kg.
About Innovent.
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Program-related investment estimated at up to US$65M (~AU$90M) for clinical costs associated with the development of the therapeutic products in the Territory, to align with Telix’s global clinicaldevelopment programs.
6 Globocan 2018 [link]
7 Chang L, et al.
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where he developed therapeutics to treat orphan eye diseases. Escolar was the principal medical consultant for Genzyme’s pivotal trial for Myozyme, an enzyme replacement therapy approved to treat adult-onset Pompe disease. since May 2018. Additionally, Viridian named Barrett Katz as its chief medical officer.
On 14 May 2018, the Company announced a collaboration with GlobalCo to work on its CDX antibody. This additional extension of the collaboration will allow us to choose the best CDX antibody for pre-clinical and clinicaldevelopment.” ” Market Abuse Regulation (MAR) Disclosure.
Onivyde, in combination with fluorouracil and leucovorin, was previously approved for the treatment of patients with metastatic adenocarcinoma of the pancreas after disease progression following gemcitabine-based therapy. Previously, the company announced positive results of the previous Phase IIa induction study in UC in September 2018.
Asthma is a chronic, inflammatory, fluctuating respiratory disease 1 that affects as many as 339 million adults and children worldwide, 1 including over 25 million in the US. 4 These patients are at risk of severe exacerbations regardless of their disease severity, adherence to treatment or level of control.
Without treatment, infants do not achieve these milestones in the natural history of the disease. More than 5,000 patients have now been treated worldwide with Evrysdi in clinical trials, compassionate use or real-world settings. About SMA SMA is a severe, progressive neuromuscular disease that can be fatal.
UC is a chronic inflammatory disease of the large intestine, also referred to as the colon, that affects the lining of the colon and may cause small sores, or ulcers, to form. “People living with UC often struggle to effectively manage recurring flare ups of the disease,” said Lotus Mallbris, M.D., About Ulcerative Colitis.
5 The European Centre for Disease Prevention and Control (ECDC) currently recommends TBE vaccination for people who live in or are traveling to these risk areas. For many years, our TBE vaccine has helped protect millions of people in Europe from this potentially serious disease. Last updated: November 2018. References.
Cadavid will be responsible for the strategy, direction and execution of the company’s clinicaldevelopment programs. Prior to Fulcrum, Cadavid held several leadership positions at Biogen, including Senior Medical Director of the multiple sclerosis clinicaldevelopment group.
Likely linked to this are the scaffolds taken through to clinicaldeveloped, since a feature common to the vast majority of CYP2D6 substrates is a basic nitrogen atom and a planar aromatic ring that need to fit in a rather buried active site, compared to the larger and more flexible active site of CYP3A4. Sugano, 2018.
The submission includes data from the ENLIGHTEN clinicaldevelopment program in schizophrenia, and pharmacokinetic (PK) bridging data comparing ALKS 3831 and Zyprexa (olanzapine), to support an indication for schizophrenia. Celgene acquired it in 2018 from bluebird bio, who originally developed the therapy.
Patients treated with this combination had a decrease in risk of disease progression or death by 48% (HR=0.52, p=0.000002) compared to patients treated with placebo and rituximab, with a median PFS of 21.5 Non-Hodgkin’s Lymphoma (NHL) comprises a highly heterogeneous group of chronic diseases with poor prognosis. months versus 13.8
Structure 3:1031-1039 (1995)
3 Waters J, Biocentury ; October 26, (2018)
4 NCT02273960; ClinicalTrials.gov ; “Study to Evaluate Safety and Efficacy in Adult Subjects With ITP (ITP)”; results posted April 1, 2019, updated July 29, 2019 and accessed Jan 11, 2021
5 Ferrant JL et al., 1 Lederman, S. International Immunol. (11):1583
The EC’s decision is based on results from the Phase 2 CheckMate -142 trial in which Opdivo plus Yervoy demonstrated a clinically meaningful improvement in objective response rate (ORR) in patients with MSI-H/dMMR mCRC who received prior treatment with fluoropyridine, oxaliplatin and irinotecan. Waxman, M.D.,
Most treatments involve focused beams of radiation or implanted radioactive seeds, both of which are “spot” treatments typically for disease that has not spread widely. . Today, approximately two-thirds of all cancer patients receive some form of radiation during the course of therapy. A foundational pillar.
Associate Professor of Medicine at UCLA, Co-director of the Gastrointestinal Oncology Program and Director of Early Phase Clinical Research at the Jonsson Comprehensive Cancer Center.
ORR (measurable disease at baseline).
Wainberg, M.D.,
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Lung cancer is the leading cause of cancer-related death in China, and with NSCLC comprising the most common form of the disease, there is significant patient need. Complete approval for these indications is contingent upon results from ongoing randomized, controlled confirmatory clinical trials. Accessed December 2020. v Zhang, T.,
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