Flagship Pioneering launches Omega Therapeutics; EU nod to Vitrakvi; Bluebird Bio reveals positive results of Starbeam clinical trial

Flagship Pioneering has launched Omega Therapeutics with an aim to take forward the genomic medicine.

The launch of Omega therapeutics was planned to focus on epigenetic drugs to target diseases. Study of changes in gene expressions without any changes in the DNA sequence is known as Epigenetics.  This often includes the addition of extra molecules that control the expression of genes.

Omega therapeutics has launched an exclusive platform that adjusts Insulated Genomic Domains (IGDs) of the human genome. Many diseases, including cancer, heart disease, diabetes, and mental illnesses are influenced by epigenetic mechanisms, and epigenetic therapy offers a potential way to influence those pathways directly.

At present, there exist only a few epigenetic drugs including Zolinza (vorinostat) developed by Merck for the treatment of Lymphoma, Otsuka Pharmaceutical’s Dacogen (decitabine) for myelodysplastic syndrome, Novartis’s Farydak (panobinostat) for multiple myeloma and Vidaza (azaacitidine) of Pharmion for myelodysplastic syndrome.

These drugs target enzymes that are involved in the regulation of gene expression.

The European Medicines Agency has given its recommendation for Bayer’s and Lilly’s Vitrakvi for the treatment of NTRK fusion-positive cancers.

Vitrakvi (larotrectinib) has received FDA’s nod last year in the month of November. It is a drug which targets the tumour based on genetic markers for cancers that are a result of a genomic abnormality called NTRK fusion.

Originally co-developed by Bayer and Loxo Oncology, the latter which was afterwards acquired by Lilly for an amount of USD 8Billion.

Vitrakvi is a second drug after Merck’s Keytruda that is tumour-agnostic. Roche’s Rozlytrek (entrectinib) became the third drug to get approval from the FDA to target NTRK fusion and ROS1 in lung cancer.

Bluebird Bio, a biotechnology firm has reported positive results from the Phase II/III clinical trials of Lenti-D gene therapy to target cerebral adrenoleukodystrophy (CALD).

CALD, a rare genetic disorder can result in severe loss of neurologic function and death.

The Starbeam trial, intended to gauge the safety and efficacy of Lenti-D in boys aged 17 and under. Its primary efficacy endpoint is the proportion of patients alive and free of major functional disabilities (MFDs) at 24 months.

The results were primarily based on the proportion of patients alive and free of major functional disabilities (MFDs) at 24 months.  

The data revealed that 88% of patients who underwent therapy and stayed under supervision for 24 months were free of major functional disabilities (MFDs) at two years and remained the same for up to five years.

MFDs are a group of symptoms comprising loss of communication ability, total incontinence, and complete loss of voluntary movement, the need for tube feeding, cortical blindness and wheelchair dependence. The company is also running observational study of allogeneic hematopoietic stem cell transplant (allo-HSCT) in CALD patients which gave favourable results.