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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.

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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 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.

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Actithera draws new investors to radiopharma drug pitch

BioPharma Drive: Drug Pricing

Daily Dive M-F Commercialization Weekly Every Wednesday Gene Therapy Weekly Every Thursday Emerging Biotech Weekly Every Tuesday By signing up to receive our newsletter, you agree to our Terms of Use and Privacy Policy. in 2013 — the field has taken off since the success of Novartis’ prostate cancer treatment Pluvicto.

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Eli Lilly to Buy Gene Therapy Firm Verve in $1 Billion Deal to Develop Heart Drug

Drugs.com

The deal gives Lilly a potential new treatment for heart disease, The Wall Street Journal reported. WEDNESDAY, June 18, 2025 — Drugmaker Eli Lilly plans to buy Verve Therapeutics, a gene-editing startup, for about $1 billion upfront.

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New tRNA tech aims to rewrite rare disease treatment

Drug Target Review

“This experience underscored the gaps in rare disease innovation, motivating me to lead Alltrna, a company pioneering engineered tRNA therapies to address a broad range of genetic diseases in a profound way unlike any other genetic modality.”

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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.