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Executive summary: Scale-up strategies for late-stage cell therapies
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Gene Therapy
Gene Therapy is the introduction of normal genes into cells in place of missing or defective ones in order to correct genetic disorders.
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Five recent cell and gene therapy discoveries
This article highlights five of the latest findings using cell and gene therapy techniques that could be used in the development or design of new therapies.
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Drug Target Review’s top content picks from 2022
In this article, Drug Target Review’s Izzy Wood and Ria Kakkad share some of the most ground-breaking moments from drug discovery this year.
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Novel gene therapy offers potential treatment for FCMD
Japanese researchers proposed using RNA interference for correcting a mistake in the genetic code of patients with Fukuyama Muscular Dystrophy (FCMD).
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Misfolding proteins bring caution for gene therapies for haemophilia
US researchers have discovered a link between protein misfolding and liver cancer, that could help improve gene therapy for haemophilia.
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P53 could be key to therapies for salivary gland cancer
Mouse models show that activating a non-mutated form of the gene could lead to developing therapies for salivary gland cancer.
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CAR-T therapy becomes a powerful tool against an autoimmune disease like MS
US researchers have shown that CAR-T therapy can be used to eliminate unwanted cells that cause autoimmunity.
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Drug Target Review Cell and Gene Therapy Supplement 2022
This supplement focuses on how cell and gene therapy can target diseases such as cancer and reduce elevated lipoprotein(a).
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CIRM grant $4.8m to fund gene therapy for rare disease
CIRM grant will fund novel gene therapy that aims for single lifelong treatment of Friedreich’s ataxia, a progressive neuromuscular disorder; a second CIRM grant will advance efforts to leverage UC San Diego research on another rare disease
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New target in the fight against heart disease
US scientists have new insight into how heart cells enable unhealthy growth, and identify a new target to intervene against heart disease.
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Drug Target Review – Issue 4 2022
A new Drug Target Review issue is now ready to download! This issue features articles which explore how artificial intelligence can enhance screening and ways to find new hits through simultaneous orthogonal screens. Also included are articles on CRISPR, immuno-oncology and RSV vaccines.
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Researchers have engineered AAV vectors that cross the blood-brain barrier
In primate models, researchers have successfully shown that developed AAVs can cross the blood-brain barrier, which keeps many drugs from getting into the brain.
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Potential treatment for neurological disorders targets brain cells
UCL researchers have developed a possible new treatment for neurological and psychiatric diseases, that works by reducing the excitability of overactive brain cells.
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New control system created for synthetic genes
MIT researchers have developed a technique that could help the production of monoclonal antibodies and other useful proteins.
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Decibel Therapeutics submits IND application for hearing loss gene therapy candidate
Decibel Therapeutics has submitted an IND application for a Phase I/II trial of DB-OTO, a gene therapy candidate to restore hearing.
