Gen­Sight has a gene ther­a­py mys­tery on its hands, which is about the last thing it want­ed at the end of Phase III.

As­sess­ing the 48-week da­ta from its first piv­otal test of a treat­ment for Leber Hered­i­tary Op­tic Neu­ropa­thy — or LHON, a rare ge­net­ic dis­ease that caus­es blind­ness — in­ves­ti­ga­tors pulled out a mean­ing­ful im­prove­ment in sight in their one treat­ed eye.  

Bernard Gilly

The mys­tery: The oth­er eyes that re­ceived a sham ther­a­py did just as well with their eye­sight. Ex­act­ly as well.

That makes the Phase III study of GS010 a fail­ure, and rather than stay to try and solve the mys­tery, in­vestors bailed, dri­ving down the Eu­roNext stock $SIGHT down by 34%.

One of the rea­sons why the ear­ly gene ther­a­py pi­o­neers like Gen­Sight picked hered­i­tary eye dis­eases to go af­ter first is that the eye is a con­tained unit, lim­it­ing off-tar­get ef­fects. But Gen­Sight’s Paris-based CEO Bernard Gilly says that as on­ly a frac­tion of pa­tients his­tor­i­cal­ly see a spon­ta­neous im­prove­ment in eye­sight, he’s won­der­ing if the ther­a­py in­ject­ed in­to one eye de­liv­ers an un­ex­pect­ed ben­e­fit to the oth­er.

At the same time, Gen­Sight tout­ed a hit on a reti­nal bio­mark­er for the dis­ease, which they claimed as ev­i­dence that the ther­a­py had an im­pact sep­a­rate from the sham.

“The fact that struc­tur­al mea­sures of the reti­na showed such a large sta­tis­ti­cal dif­fer­ence with treat­ment is com­pelling and ob­jec­tive ev­i­dence that this gene ther­a­py pro­tects the in­tegri­ty of many reti­nal gan­glion cells from the dam­age of LHON,” com­ment­ed com­pa­ny co-founder José-Alain Sa­hel.

That’s a messy ar­gu­ment, with the com­pa­ny ar­gu­ing that it ap­peared the ther­a­py im­proved sight for both eyes, but on­ly hit the sec­ondary bio­mark­er in the treat­ed eye. 

The com­pa­ny — which tried and failed to make it on­to Nas­daq — will be left look­ing for more da­ta to an­a­lyze from its next two Phase III stud­ies, which are up­com­ing. But easy ex­pla­na­tions will be hard to come by.

Not even one month after Big Pharma took notice of Artiva when Merck signed a collaboration worth nearly $2 billion in milestones, the off-the-shelf NK cell biotech already has its next big fundraise.

Artiva returns from the venture well Friday with a $120 million Series B round, money they will use to get their first program into the clinic and to file INDs for another two candidates. The raise marks the latest development in a rapidly expanding footprint for Artiva, which, in addition to the Merck deal last month, has now raised almost $200 million since its Series A last June.

The Endpoints News team is continuing to track IPO filings for 2021, and we’ve designed a new tracker page for the effort.

Check it out here: Biopharma IPOs 2021 from Endpoints News

You’ll be able to find all the biotechs that have filed and priced so far this year, sortable by quarter and listed by newest first. As of the time of publishing on Feb. 25, there have already been 16 biotechs debuting on Nasdaq so far this year, with an additional four having filed their S-1 paperwork.

Biotech Voices is a collection of exclusive opinion editorials from some of the leading voices in biopharma on the biggest industry questions today. Think you have a voice that should be heard? Reach out to senior editors Kyle Blankenship and Amber Tong.

We often think a person’s transition from a healthy to a diseased state is binary. But that’s often not the case. In reality, the onset of a disease is not something that occurs overnight, and the majority lie on a continuum that is impacted by a multitude of factors. Some of these factors are in a patient’s control. Others are not.

This is the case in nonalcoholic fatty liver disease (NAFLD) and nonalcoholic steatohepatitis (NASH), two of the most complex diseases that “live” on this proverbial continuum. The clinical onset of NAFLD — and ultimately NASH — is a complex process that is closely related to obesity, insulin resistance and impaired adipose tissue metabolism.