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Home » Topics » Drugs » Gene therapy

Gene therapy
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Precision targeted therapy concept illustrated by dart hitting bullseye of target

Is APAC ready for precision medicine?

Aug. 12, 2025
By Tamra Sami
No Comments
Precision medicine is becoming a reality in Asia Pacific as more targeted therapies are being developed that are tailored to individual patients, offering a potential cure for disease. But is Asia Pacific ready to harness this transformation, and if not, what are the hurdles that need to be cleared?
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Stock prices with loss/gain arrows

A mild market reaction for companies on Prasad’s return

Aug. 11, 2025
No Comments
After about a two-week absence as the U.S. FDA’s CBER director, Vinay Prasad’s return overall prompted a mild reaction on Wall Street for some stocks tied to companies developing cell and gene therapies.
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Precision targeted therapy concept illustrated by dart hitting bullseye of target

Is APAC ready for precision medicine?

Aug. 6, 2025
By Tamra Sami
No Comments
Precision medicine is becoming a reality in Asia Pacific as more targeted therapies are being developed that are tailored to individual patients, offering a potential cure for disease. But is Asia Pacific ready to harness this transformation, and if not, what are the hurdles that need to be cleared? A new report by LEK Consulting examines Asia Pacific’s readiness for what it calls the “Precision Era,” and examines four key biopharma markets in Australia, China, Japan and South Korea.
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DNA double helix under a magnifying glass
Nephrology

Purespring gets go-ahead for first kidney-directed gene therapy

Aug. 5, 2025
No Comments
Purespring Therapeutics Ltd. has received the go-ahead for a phase I/II trial of its investigational gene therapy PS-002 from both the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) and the National Health Service Health Research Authority and Research Ethics Committee.
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Illustration of kidneys with DNA double helix
Nephrology

Non-viral gene therapy to treat cystinuria

Aug. 4, 2025
No Comments
In an effort to develop next-generation treatments for cystinuria, researchers at Vanderbilt University and Tennessee Valley Health Services have used a non-viral piggyBac transposon approach to insert the Slc3a1 gene into one kidney of mice lacking the endogenous gene, which services as a model of type A cystinuria.
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CBER departure spurs Street celebration, ACIP investigation looms

July 30, 2025
By Mari Serebrov
No Comments
The news that Vinay Prasad has stepped down as CBER director at the U.S. FDA had some biotech stocks literally jumping in joy as the market opened July 30. Meanwhile, Prasad’s decisions regarding vaccine development, as well as actions by Makary and HHS Secretary Robert Kennedy, are coming under fire.
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Illustration of gene editing in neurons

Prime editing could cure a rare childhood hemiplegia disorder

July 30, 2025
By Mar de Miguel
No Comments
An experimental gene therapy based on the prime editing technique could become an effective treatment for alternating hemiplegia of childhood, a severe and currently incurable rare disease. David Liu’s lab at the Broad Institute, the inventor of this gene edition methodology, together with scientists from The Jackson Laboratory, successfully reversed the effects of five mutations associated with this disorder in a mouse model.
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Regulatory upswing for Sarepta seesaw with Elevidys in DMD

July 29, 2025
By Randy Osborne
No Comments
Sarepta Therapeutics Inc.’s adventure with the Duchenne muscular dystrophy (DMD) AAV-based gene therapy Elevidys (delandistrogene moxeparvovec) continued as the firm said it would restart shipments of the compound for ambulatory patients “imminently,” with the U.S. FDA’s blessing.
Read More
Illustration of gene editing in neurons
Drug design, drug delivery & technologies

Prime editing could cure a rare childhood hemiplegia disorder

July 29, 2025
By Mar de Miguel
No Comments
An experimental gene therapy based on the prime editing technique could become an effective treatment for alternating hemiplegia of childhood, a severe and currently incurable rare disease. David Liu’s lab at the Broad Institute, the inventor of this gene edition methodology, together with scientists from The Jackson Laboratory, successfully reversed the effects of five mutations associated with this disorder in a mouse model.
Read More
Silhouette of child and brain

Rett bet set includes Neurogene, Taysha gene therapies

July 24, 2025
By Randy Osborne
No Comments
Neurogene Inc.’s disclosure June 30 of its registrational trial design in Rett syndrome (RS) added spice to the space, where Taysha Gene Therapies Inc. is another closely watched player. The New York-based firm said Embolden will test NGN-401, a gene therapy for the treatment of females age 3 and older with RS. U.S. regulators have signed off regarding key aspects of Embolden’s structure.
Read More
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