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Gene editing extends lifespan in mouse model of prion disease

Broad Institute

In 2018, Liu, who works on the same floor as Minikel and Vallabh at Broad, approached them and proposed a collaboration. Theres still a long way to go to make this a therapy, Minikel said. Tags: Neurodegeneration Rare Disease Gene therapy Minikel remembers thinking, Theres something really promising there. Nature Medicine.

Disease 144
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Aleta’s Novel T Cell Engagers

SugarCone Biotech

When we look at how TCEs interact with targeted cancer cells we can quickly see how cancers respond to T Cell-based therapies. The most common response is quite expected, which is that the cancer cells escape from TCE therapy by downregulating expression of the targeted cancer protein (Ref. Lancet Haem 11: e693-e707. Shen et al.

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Automated red blood cell exchange: bridging treatment gaps in sickle cell disease care

Drug Target Review

Despite recent advances in gene therapy for sickle cell disease (SCD) , automated red blood cell exchange (aRBCX) remains a cornerstone therapy that plays a vital yet underutilised role in managing complications and enhancing quality of life for millions living with this devastating condition worldwide.

Disease 52
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Bayer Expands Collaboration with Tsinghua University to Boost Pharma R&D in China

The Pharma Data

Oncology remains a core priority, reflecting the rising global burden of cancer and the need for more effective, targeted therapies. The goal is to transform laboratory discoveries into clinically relevant therapies that can reach patients faster and more efficiently. Source link

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Building better brain models for Parkinson’s disease and beyond

Drug Target Review

In this interview, Professor Schwamborn discusses his research, the promise of brain organoids, and his vision for the future of stem cell-based therapies. ” By using personalised models, researchers can identify subgroups of patients with similar molecular characteristics, potentially leading to the development of targeted therapies.

Disease 52
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Inavolisib

New Drug Approvals

3] Primary endocrine resistance was defined as relapse while on the first two years of adjuvant endocrine therapy (ET) and secondary endocrine resistance was defined as relapse while on adjuvant ET after at least two years or relapse within twelve months of completing adjuvant ET. [3] New Drug Therapy Approvals 2024 (PDF).

FDA 57
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Elacestrant 

New Drug Approvals

Eligible participants had experienced disease progression on one or two prior lines of endocrine therapy , including one line with a CDK4/6 inhibitor , and could have received up to one prior line of chemotherapy in the advanced or metastatic setting. [2] 1] [4] It is taken by mouth. [1] 3] [7] PATENTS Cruskie MP, et al.

FDA 62