Ann: ATL1102 for DMD EMA PIP feedback and FDA regulatory progress, page-49

PS: we know Gene Therapy has had a crappy past 12 months or so - here's another failure.

This time our Swiss McDuck yet again - Roche - sees another of its GT investments fail.

@waynesworld

Here is that pesky "immune response" issue appearing....and "inflammation" as adverse effect.

Roche walks away from 4DMT's eye gene therapy before early-stage trial finishes
by Kyle LaHucik |
Jun 24, 2021 4:10pm

Roche has told 4D Molecular Therapeutics that it's time to wind down a sponsored trial for an eye disease gene therapy and send the rights for the therapy back to the small biotech.

4DMT vowed to roll on with developing the therapy, even if Roche is questioning the risk-benefit profile after reviewing early data.

The Swiss Big Pharma had been funding an early stage trial for 4D-110 in patients with advanced choroideremia, a blinding disease with no approved therapies. That deal will now terminate in September, with the full rights for the therapy transferring back to 4DMT.
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Roche may have cut the cord based on early preclinical data that showed poor delivery of the payload and the fact that 4DMT's gene therapy uses a vector known to spur immune responses, Evercore said.

Another reason for Roche's exit may have been Adverum Biotechnologies' recent shaky safety results and a Biogen trial failure earlier this month. Those events have put a damper on the entire gene therapy field in eye diseases.
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4DMT also disclosed its own set of adverse events, although not nearly as concerning as the blinding event seen in the Adverum trial. Half of the patients who received 4D-110 reported eye inflammation.
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The news comes six months after 4DMT raised $222 million in an IPO and added a clinical leader for ophthalmology.

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