A kink in the gene therapy supply chain

Gene therapy is increasingly proving its mettle in repairing faulty DNA — but can manufacturers meet patient demand? The process of engineering the viral vectors that ferry restorative genes into a patient’s cells remains cumbersome and costly.

Until now, gene therapy companies like Biomarin have relied on academic research centers or contract manufacturers to churn out the necessary viral bits. But as the market heats up for these therapies, companies are trying to figure out how to make these complex genetic parcels in-house.

“It’s not like making penicillin,” one gene therapy exec told STAT. “We don’t even have the technology to take two batches side-by-side and see if they’re the same product.”

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New rules may dampen Chinese investment in biotech

U.S. biotech is funded increasingly by Chinese investors. But the Trump administration issued a new set of rules last month that call for additional scrutiny of some foreign investments — leading many in biotech to wonder whether this might slow the flow of Chinese dollars into the sector. What’s unclear, however, is how these new regulations will be enforced. 

STAT’s Rebecca Robbins and Ike Swetlitz took a deeper look into the kinds of investments, companies, and products that are likely to be impacted by these rules — and what the new rules might look like from abroad. 

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PARP for Parkinson's?

PARP inhibitors, or drugs that block the enzymes involved in DNA repair, have shown a lot of promise in treating a wide range of cancers. But companies like Tesaro and Clovis Oncology — which already have secured approval for their respective PARP inhibitors — ought to take note of this new study in Science. 

The authors describe a new molecular pathway that might account for why Lewy bodies — deposits of certain proteins in Parkinson’s — might build up. Basically, a protein called a-synuclein — as seen in a test tube, anyway — can activate these PARP repair proteins, which in turn create highly pathogenic fibers. When they are implanted into cell cultures, or the brains of mice, they lead to protein buildup and neuronal death. 

The basic biology here suggests that PARP inhibitors, such as those being developed for a wide slew of cancers, might have some efficacy in slowing the accumulation of protein tangles in the Parkinson’s brain.

Sage got (almost) everything it wanted

A day after a bruising nine-hour session with Alkermes, the FDA’s advisory panel of depression experts had a much more tranquil experience on Friday with Sage Therapeutics.

The group voted 17-1 that the benefits of Sage’s treatment for postpartum depression, called brexanolone, outweighed the drug’s risks. That makes it a virtual lock to win FDA approval next month, but the good news comes with a caveat: The panel wasn’t won over by the idea of allowing Sage’s drug, which requires a 60-hour infusion, to be in administered in patients’ homes. If the FDA agrees and restricts brexanolone’s use to within hospitals and clinics, it could limit the treatment’s commercial potential.

Most relieving to Sage’s investors is that the day-long session didn’t cast any doubt on SAGE-217, an in-development treatment that promises to match brexanolone’s effects but in pill form. Analysts say SAGE-217 could be a multibillion-dollar drug, and while that depends on trials still underway, brexanolone’s day at the FDA did nothing to raise doubts.