Michael Ehlers announces plans to start next ‘signature’ genetic medicine company with $ 75 million from Apple Tree Partners – Endpoints News

When Michael Ehlers left the ship at Biogen to become a venture capital partner and CSO at Apple Tree, he also got a new job as CEO of a gene therapy startup named Limelight Bio. Stealth biotechnology is now poised to shed some – but not many – of its sails.

Seth harrison

As ATP Managing Partner Seth Harrison says, the new company grew out of a strong relationship with co-founders, Jean Bennett and Phil Johnson. Bennett, who led the early research into the historic gene therapy known as Luxturna alongside Spark Therapeutics CSO Kathy High, now heads Penn’s Center for Advanced Retinal and Ocular Therapies. Meanwhile, Johnson left his post at Children’s Hospital of Philadelphia to become the Limelight CSO.

“Initially, we were focused on supporting Jean and CAROT and some retinal dystrophy gene therapy projects using standard AAV approaches,” he said, “but as we learned Knowing the wealth of technologies that could be accessed through Jean and Phil, some from Penn, some not, we were very excited about the potential for building a great gene therapy business.

Jean Bennett (via Drexel)

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Limelight pursues gene therapy in its broadest sense. In addition to the standard gene replacement delivered by an adeno-associated virus, or AAV, biotechnology will also explore non-viral approaches and what they call multi-exon gene correction.

The wide range of targets and modalities that Limelight seeks makes it unique, said Bennett, who also co-founded ophthalmic gene therapy company GenSight. Some of the ultra-rare diseases they want to treat would have been ruled out by most companies, she said. Terminal news.

The potential of this suite of technologies to move the needle of genetic medicine and expand it to new diseases and geographies was also what drew Ehlers to this opportunity in the first place. ATP’s promise of substantial capital to deploy in space probably didn’t hurt either.

“The degree of effectiveness that you can get from these therapies is substantial, it’s not just incremental effectiveness, you squint and find a p-value,” he said.

Phil johnson

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With well-known gene replacement technology, Limelight boasts of sophisticated methods that allow them to insert larger genes than before and better control gene expression. When it comes to gene correction, Ehlers is careful to distinguish what they do from the “classic old typewriter” effect typically associated with gene editing.

“We use the term ‘correction’ explicitly because we are able to do so much more than a small dot of the gene at a time,” he said. “We can make large portions at a time, at endogenous levels – and not requiring exogenous enzymes or cofactors” such as Cas9.

He also suggested that their technology does not carry the risk of permanent genomic alteration, hinting at reversible RNA or DNA changes.

Exactly how Limelight will accomplish these feats, or how many employees work on the technology platforms, or what manufacturing operations they have in place, Ehlers isn’t quite ready to divulge, repeatedly saying to “stay at it. listening “.

He is eager to preview, however, that Limelight expects to be a clinical venture within one to two years, ushering in at least two programs in retinal diseases. This will be followed by programs on hematological / metabolic, neurological and auditory disorders – each on indications they have identified with the fastest path to clinical and regulatory validation.

As Limelight speaks on the same day that former Ehlers colleagues at Biogen present one of the year’s most anticipated biotech datasets at an Alzheimer’s disease conference, There is a question that at least one analyst has asked out loud: How in the world would he have left Biogen if he had believed in aducanumab?

He won’t.

“My main focus is really how excited I am about Limelight and the promise of truly building a signature gene therapy company,” Ehlers said.

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