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Infant with rare, incurable disease is first to successfully receive personalized gene therapy treatment

Science Daily: Pharmacology News

A research team has developed and safely delivered a personalized gene editing therapy to treat an infant with a life-threatening, incurable genetic disease. The infant, who was diagnosed with the rare condition carbamoyl phosphate synthetase 1 (CPS1) deficiency shortly after birth, has responded positively to the treatment.

Treatment 321
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Novel stem cell therapy repairs 'irreversible' corneal damage in clinical trial

Science Daily: Pharmacology News

Results from a phase 1/2 clinical trial of a novel stem cell treatment for cornea injuries found 14 patients treated and tracked for 18 months had a more than 90% success rate at restoring the cornea's surface and improvements in vision.

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New gene therapy for muscular dystrophy offers hope

Science Daily: Pharmacology News

A new gene therapy treatment for Duchenne muscular dystrophy (DMD) shows promise of not only arresting the decline of the muscles of those affected by this inherited genetic disease, but perhaps, in the future, repairing those muscles.

Therapies 310
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Breast cancer treatment advances with light-activated 'smart bomb'

Science Daily: Pharmacology News

Scientists have developed new light-sensitive chemicals that can radically improve the treatment of aggressive cancers with minimal side effects. In mouse tests, the new therapy completely eradicated metastatic breast cancer tumors.

Treatment 301
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Pfizer backs CRISPR biotech Caribou in latest cell therapy investment

BioPharma Drive: Drug Pricing

Five years after helping launch Allogene Therapeutics, Pfizer is backing another developer of “off-the-shelf” cell therapies in a deal that gives the drugmaker the chance to acquire a multiple myeloma treatment.

Therapies 333
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Current Challenges With Highly Active Antiretroviral Therapy and New Hope and Horizon With CRISPR‐CAS9 Technology for HIV Treatment

Chemical Biology and Drug Design

CRISPR-CAS9 is an innovative gene editing technology to precisely remove or incorporate into the gene of interest; CRISPR-CAS9 shows promising results in HIV treatment. This article outlines the current antiretroviral therapy and its adverse effects but also CRISPR/Cas technology.

Treatment 100
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New cell therapy model accelerates cancer treatment development

Drug Target Review

In the rapidly advancing field of cell therapies, Dr Jason Bock has emerged as a leader, known for his innovative approach to optimising the development process. With over 25 years of experience in therapeutics, Bock has played a pivotal role in shaping the future of cell therapies, particularly through his work at CTMC.