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STAT+: Pharmalittle: Pfizer scaling back early-stage research into rare diseases; BioNTech and U.K. partner on cancer trials

STAT News

… Pfizer told employees it plans to pull back on early-stage research into treatments for rare diseases, including the development of new viral-based gene therapies , Barron’s reports. Among the assets on the chopping block is a gene therapy manufacturing facility in Durham, N.C.,

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HFpEF vs. HFrEF: How To Improve Heart Failure Drug Development

XTalks

Late-stage clinical trial failures are largely to blame for the lack of effective treatment options for patients with HFpEF, and poor five-year survival rates make this an area of great unmet need. Heart failure affects about 6.2 Figure 1: The use of Mendelian randomization to validate genetic drug targets.

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Many children with cardiomyopathy have a genetic mutation but few are screened

Scienmag

— A national, University at Buffalo-led study on genes in pediatric cardiomyopathy demonstrates strong evidence for routine genetic screening in children with the disease. The study, published April 28 in […].

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Bayer delivers on medical innovation fueling transformation of pharma business

The Pharma Data

Our leadership in cardiology, radiology and women’s health is recognized worldwide, and we are expanding our presence in oncology, working tirelessly to bring forward new approaches that can change the treatment paradigm for patients,” said Stefan Oelrich, Member of the Board of Management, Bayer AG and President of Bayer’s Pharmaceuticals Division.

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BlueRock Therapeutics Announces Completion of Enrollment of Phase 1 Trial in Patients with Parkinson’s Disease

The Pharma Data

the completion of enrollment of its Phase 1 (Ph1), open-label trial of pluripotent stem cell-derived dopaminergic neurons in patients with Parkinson’s disease (PD). Ph1 clinical trial is to evaluate the safety, tolerability, and?preliminary?efficacy?of Senior Vice President, Clinical Development, BlueRock Therapeutics.

Trials 52
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Pfizer Investor Day Features Significant Number of Pipeline Advances for COVID-19 Programs and Across Numerous Therapeutic Areas

The Pharma Data

The company announced the initiation of its Phase 1b clinical trial to evaluate the safety of a novel investigational therapeutic for COVID-19, PF-07304814. Based on these data, the Company plans to initiate the pivotal study in the next several weeks, with the plan to perform an interim analysis of the clinical data in 2022.

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Evinacumab could help some patients with severe hypertriglyceridemia

Scienmag

Patients’ genetic profile strongly influences size of effect in early-phase trial People with extremely high levels of triglycerides (a type of fat in the blood) and a specific genetic profile saw a substantial reduction in triglycerides after taking the human monoclonal antibody evinacumab compared with those taking a placebo, in a study presented (..)