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Sarepta Stock: a promising gene therapy against muscular dystrophy

Therapeutic Sarepta (SRPT) came closer to hatching Wednesday after its gene therapy has shown promise in the treatment of a form of muscular dystrophy.


Today, Sarepta's shares have risen 8.3% to 151.68, more than triple their average volume. The shares are approaching a point of purchase at 169.97 on a cup base that began to form in early October.

Gene therapy has been tested in three patients with a form of muscular dystrophy belts. In this form of muscular dystrophy, patients lack a protein called beta-sarcoglycan. Sarepta gene therapy, known as MYO-101, attempts to encode this protein.

The patients underwent a biopsy two months after the treatment. On average, protein levels were 51% above normal levels. Sarepta only needed to show that 20% of this protein in the patients' muscles was effective. The drug, if approved, would be the first treatment of this form of muscular dystrophy.

"The positive results of our first MYO-101 cohort reinforce our determination to build our gene therapy engine quickly and efficiently," Sarepta CEO Doug Ingram said in a written statement.

Promise of gene therapy in muscular dystrophy

All patients showed a drop in creatine kinase levels, Sarepta said in a press release. This is an enzyme that can show muscle damage. On average, creatine kinase levels dropped by 90%.

According to the Mayo Clinic, two patients had elevated liver enzymes, which may indicate inflammation or damage to liver cells. This can be a side effect of gene therapy. Both patients received additional treatment with steroids and the symptoms disappeared.

Brian Abrahams, an analyst at RBC Capital Markets, says the data released Wednesday "significantly disrupts" gene therapy.

Protein "Expression data has been met or exceeded (key opinion leader) and company thresholds, correct location has been demonstrated and biomarkers well correlated", he stated in a report to customers.

Abrahams raised its target price on Sarepta shares from 164 to 199, while maintaining its outperformance rating. He now considers that the treatment of muscular dystrophy of the body belts is capable of generating long-term sales of $ 2.4 billion.

Gene Therapy Space Heats Up

MYO-101 is one of the many gene therapies under the Sarepta belt. Sarepta also goes against Pfizer (PFE) and Solid Biosciences (SLDB) with gene therapy to treat Duchenne muscular dystrophy. This form of muscular dystrophy is caused by the absence of another protein.

Gene therapy has been a hot topic lately. Earlier in the week, rock (RHHBY) is committed to buying Therapeutic Spark (ONCE) for 4.8 billion dollars. Spark owns the first gene therapy in the United States, called Luxturna, which treats a disorder that is evolving into blindness.

Last year, Novartis (NVS) acquired gene therapy player AveXis for $ 8.7 billion. The company hopes to gain approval for gene therapy to treat spinal muscular atrophy, another muscle disease.


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