News

The U.S. FDA has granted Capricor Therapeutics RMAT designation for its lead investigational cell therapy for the treatment of Duchenne muscular dystrophy, CAP-1002.

Fibrocell Science announced the submission of an Investigational New Drug Application with the U.S. FDA for FCX-013, a gene therapy candidate for the treatment of moderate to severe localized scleroderma.

Biotechnology company Avrobio has completed a $60 million Series B financing to advance multiple gene therapies, including AVR-RD-01, a proposed single-dose lentiviral gene therapy for Fabry disease (FD).

Rare pediatric disease designation was granted by the FDA to MeiraGTx’s A002 (ZZV2/8-hCARp.hCNGB3) for achromatopsia.

Final updated results of the pivotal phase 2 study that led to last year’s FDA approval of the first chimeric antigen receptor (CAR) T-cell therapy were published Wednesday in the New England Journal of Medicine.

Coverage from the 59th Annual Meeting and Exposition of the American Society of Hematology, December 9-12, 2017.

Chimeric antigen receptor (CAR) T-cell therapy has been named the Advance of the Year in ASCO’s Clinical Cancer Advances 2018. According to the annual report, CAR T-cell therapy is “poised to transform the outlook for children and adults with certain otherwise incurable cancers."

The annual update on oncology treatment highlights the most impactful clinical cancer research and policy developments over the past year in CAR T-cell therapy.

Jesus Berdeja, MD, discusses ongoing research with bb2121 in patients with relapsed/refractory myeloma.

This morning, Abeona Therapeutics announced that the U.S. Food and Drug Administration granted EB-101, its gene therapy for epidermolysis bullosa, Regenerative Medicine Advanced Therapy designation.

Parent Project Muscular Dystrophy announced that the first patient with Duchenne muscular dystrophy has been dosed with microdystrophin gene therapy.

Nina Shah, MD, discusses immunotherapy and cellular treatments moving fast through the multiple myeloma pipeline, and the hope for CAR T-cell therapy to move up to earlier lines of treatment.

Brentuximab vedotin (Adcetris) has been approved by the European Commission for the treatment of patients with CD30-positive cutaneous T-cell lymphoma after at least 1 prior systemic therapy.

uniQure was granted Orphan Medicinal Product Designation from the European Medicines Agency for AMT-130, an investigational gene therapy for the treatment of Huntington’s disease.

Celgene has announced plans to acquire Juno Therapeutics, maker of the CAR T-cell therapy lisocabtagene maraleucel (JCAR017), for $87 per share, totaling approximately $9 billion.

Babis Andreadis, MD, discusses the successes seen with CAR T-cell development in the past year and the use of CAR T-cell therapy in patients with NHL.

In 2018, uniQure N.V. expects to advance the clinical development of AMT-130, its investigational gene therapy for the treatment of Huntington’s disease.

The FDA has granted a priority review to the CAR T-cell therapy tisagenlecleucel for adult patients with relapsed/refractory DLBCL who are ineligible for or relapse after autologous stem cell transplant.

Researchers at St. Jude Children’s Research Hospital have developed a new gene therapy that creates fully-functioning immune systems in babies diagnosed with severe combined immunodeficiency, commonly referred to as the “Bubble Boy” disease.

Why healthy adults should still get the flu vaccine; President Donald Trump undergoes first medical checkup of his presidency; review of Luxturna finds the $850,000 gene therapy needs a price discount.

The combination of the CAR T-cell therapy axicabtagene ciloleucel and the PD-L1 inhibitor atezolizumab was highly active with a manageable safety profile in patients with refractory DLBCL enrolled in the phase I/II ZUMA-6 trial.

Mesenchymal stem cell transplantation showed long-lasting and consistent effects on opioid tolerance and opioid-induced hyperalgesia in rat and mouse models.

At the 36th Annual J.P. Morgan Healthcare Conference, uniQure announced that in 2018, the company intends to advance its gene therapy AMT-061 into a pivotal study for hemophilia B.

Eisai’s multiple receptor tyrosine kinase inhibitor lenvatinib and Merck’s anti–PD-1 therapy pembrolizumab as a combination therapy for patients with renal cell carcinoma received the designation based on results from the renal cell carcinoma cohort in Study 111.

BioMarin Chief Executive Officer Jean-Jacques Bienaime spoke about the company’s development of valoctocogene roxaparvovec, a gene therapy in development for the treatment of Hemophilia A.

The FDA has granted the combination of the PD-1 inhibitor pembrolizumab and the VEGF/FGF inhibitor lenvatinib a breakthrough therapy designation for the treatment of patients with advanced and/or metastatic renal cell carcinoma.

Global Blood Therapeutics, Inc. was granted Breakthrough Therapy Designation from the U.S. Food and Drug Administration for voxelotor for the treatment of sickle cell disease.

Last week, Pfizer announced a new partnership with Sangamo. As part of the agreement, the sides will team to develop a potential gene therapy to treat ALS, or Lou Gehrig’s disease.

While chemotherapy with thoracic radiation has been established as the standard of care for the initial treatment of non-metastatic small-cell lung cancer, a large proportion of patients do not receive these treatments and in turn have lower overall survival, according to a study published in JAMA Oncology.

Recent data have found that the same gene editing platform can disable the defective gene responsible for amyotrophic lateral sclerosis in mice. The therapy, which extended the lifespan of the mouse models by 25%, delayed the onset of the muscle wasting which characterizes the disease.