Silas Inman

The FDA has lifted a clinical hold placed on an IND for VY-HTT01 in Huntington disease, allowing Voyager Therapeutics to initiate a phase 1/2 study for the agent later this year.

A policy allowing enforcement discretion for cellular-derived therapies, including stem cell-based products, will end on May 31, 2021, allowing the FDA to take more action against unapproved regenerative medicines.

The FDA has lifted a clinical hold on the development of etranacogene dezaparvovec for patients with hemophilia B.

The gene therapy SAR439483 demonstrated promising early signs of activity in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D.

Vertex Pharmaceuticals will take the lead on worldwide development, manufacturing, and commercializing of CTX001, with payments and split economics to CRISPR Therapeutics.

The first patient has been enrolled and treated with RGX-121 in the third cohort of a phase 1/2 study for patients up to 5 years old with severe MPS II.

RP-L201 continued to show promising preliminary efficacy for patients with severe LAD-I after additional follow-up in a phase 1/2 study.

The FDA has granted an orphan drug designation to CYNK-001 as a potential treatment for adult patients with recurrent glioblastoma multiforme.

Lifileucel elicited objective responses, including complete responses, for a median duration that was not yet reached at 28.1 months for patients with advanced melanoma.

AAV2 TrkB-2A-mBDNF resulted in early signs of efficacy in preclinical studies of glaucoma and humanized tauopathy, which could be translatable to other neurodegenerative polygenic disorders.

Enrollment has completed for the phase 3 GEM-3 study for beremagene geperpavec for patients with dystrophic epidermolysis bullosa. Topline results are anticipated before the end of 2021.

Etranacogene dezaparvovec was "highly unlikely" to be the cause of hepatocellular carcinoma in a patient with hemophilia B enrolled in the phase 3 HOPE-B trial, according to an independent investigation.

The tumor infiltrating lymphocyte therapy LN-145 was feasible, safe, and effective in patients with pretreated advanced cervical cancer.

In 2021, Abeona Therapeutics is anticipating to complete enrollment in its phase 3 VIITAL study and additional findings for ABO-102 and ABO-101.

NurOwn was safe and led to improvements in function and cognition at 28 weeks compared with baseline for patients with progressive multiple sclerosis.

RP-L301 demonstrated improvements in hemoglobin at 3- and 6-months for the first 2 patients treated in a phase 1 study for pyruvate kinase deficiency.

The FDA has granted a fast track designation to the placental-derived natural killer cell therapy CYNK-001 as a potential treatment for adult patients with recurrent glioblastoma multiforme.

The lentiviral vector used to create LentiGlobin was deemed a very unlikely cause of AML in a patient with sickle cell disease.

The FDA has granted an RMAT designation to RP-L201 as a potential treatment for patients with leukocyte adhesion deficiency-I.

The FDA has granted an RMAT designation to valoctocogene roxaparvovec for hemophilia A.

UniQure plans to file etranacogene dezaparvovec with the FDA for hemophilia B in March, pending a positive resolution to a clinical hold placed in December.

Rocket Pharmaceuticals has summarized multiple major milestones for its 5 gene therapies from 2020 and provided a forecast and outlook for what is expected to be a banner year for the organization.

Preliminary evidence pinpoints classical causes for AML in patient treated with LentiGlobin in a phase 1/2 study for sickle cell disease.

Sanofi has entered a new agreement with SIRION Biotech to develop tissue-selective AAV vectors for novel gene therapies.

An independent board has determined that ofranergene obadenovec is safe allowing the phase 3 OVAL study to continue.

The suspension is due to recently reported diagnoses of AML and myelodysplastic syndrome in 2 treated patients.

The CAR T cell therapy bb21217 demonstrated high very good partial response or better rates in patients with heavily pretreated relapsed/refractory multiple myeloma.

Treatment with the anti-CD19 CAR T-cell therapy KTE-X19 elicited a complete remission rate of 67% and an objective response rate of 93% for patients with relapsed/refractory mantle cell lymphoma.

The CAR T-cell therapy axicabtagene ciloleucel (axi-cel; Yescarta) induced a median overall survival of 25.8 months for patients with refractory large B-cell lymphoma.

An investigational new drug application for the therapy, which is labeled FT596, was approved in September 2019 and human trials are scheduled to begin in the first quarter of 2020.