RGNX Regenxbio Inc

$RGNX Rocket's call was interesting in the amount of flexibility the FDA is showing for their therapy, this therapy mind you had an SAE that resulted in a death. Apparently the FDA is pushing Rocket to start a trial in female danon patients ASAP and there's a world where it gets approved off of 7 patients. From my perspective this means we will probably get decent flexibility on how 121 resolves assuming it isn't actually causing really bad stuff in the spine. For 202 I continue to fail to see how it is not approved assuming the safety and efficacy have not meaningfully degraded.

$RGNX If these rates of DRSS improvement pull through into the phase 3 I just don't see how SCS 314 for NPDR is not a 5-10 billion dollar drug or even more. We also have to remind ourselves here no one else has even ran a phase 2 gene therapy trial in NPDR, the other company that could potentially pursue it in fdmt does not have the capital to do so and probably would have to start with a phase 2. By the time anyone could even think of trying to do a follower Regenx will have atleast a 3 year lead, and more likely will have 5-6.

$RGNX They put up the Baird cast aswell, so we'll see what they have to say for themselves tomorrow.

$RGNX Bottom line, ABBVIE is seeing the value of this program. I want to see what they do with the pre-clinical assets for GA that RGNX has been bragging about saying they were getting ready for an IND.

$RGNX Durability is the whole bet in gene therapy, and Regenxbio just put a three-year number on it. Phase II ALTITUDE follow-up, presented Thursday at the Retina Society meeting: one in-office suprachoroidal dose of sura-vec for non-proliferative diabetic retinopathy. A majority of Dose Level 3 patients showed a greater than two-step DRSS improvement at three years, with no vision-threatening events and no additional treatment needed. Current preventive care for NPDR is ongoing intraocular injections. A single dose that holds for three years replaces the repeat-visit treadmill. No new safety signals, and no intraocular inflammation on short-course prophylactic steroids. It carries AbbVie's ABBV-RGX-314 code, so the commercial path is not a one-biotech job. Closed Friday at 7.40, up 2.2 percent, on a market cap under half a billion. The durability read is pricing in slowly. Tracking: Phase III design and timing, now that the durability question has three years of cover.

$RGNX RE: DMD FDA application. I got from a very good source that the application will be submitted in full as a complete BLA Q1 rather than a rolling application. From HQ source.

$RGNX The new director showed some confidence with open market purchases; shareholders, not so much. Will be interesting to see if he averages down.

$RGNX this is the dose for atmosphere and ascend. Low dose(cohort 3) and high dose(cohort 4). Pretty impressive!

$RGNX How does Kodiak’s Zenkuda, affect the competitiveness of 314 ? Anybody have an opinion on thst ?

$RGNX sounds like Abbvie is excited in 314. This is their chance to recoup the $62B they spent buying Allergan which turned out a bust due to abicipar pegol failed in phase 3.

$RGNX The Jan 15, 2027 calls for $10 strike are about $1.50-1.60. If they come anywhere close to 75-80% reduced injection burden, which I do anticipate, then a 5X+ would not be out of the question. I bought some since shares would either tank or jump at the read in all likelihood. So for a buck and half, yeah, I will roll dice.

$RGNX The market makers really "eff" with this stock. Drop it 10 cents plus on what, 9k shares? Gonna be like this until the first approval hits or superior AMD news.

$RGNX usually when this stock drops like this for no reason, we see an even bigger drop the following day! Something leaked

$RGNX in Hunter syndrome, spinal cord could get damaged over time. And yet, spine MRIs are not a routine part of standard clinical trials or regular care for patients with Hunter syndrome. Tell me about it, because IMO it is impossible that there is no database of spinal MRI in Hunter syndrome general population. But FDA asks RGNX to perform spinal MRI, and then puts the drug on hold, without knowing what the prevalence of these nodules is in the Hunter syndrome general population. Either I am a clueless moron, or these health agencies and drug companies have been scamming the human species no matter what disease is in question. Sugary drinks, opioids, pesticide exposure, Alzheimer's amyloid beta hypothesis, chemotherapy poisons, mRNA vaccines, DMD rejection of CAPR, pick your favorite topic..

$RGNX Lots of great information with todays conference presentation. Especially the preparation for commercialization. Great things ahead for RGNX in 2027!

$RGNX Great conference, makes me more confident in them. Just need to wait and play the long game.

$RGNX just for fun, what do you think the SP will be at EoY? Needless to say, this year has NOT gone according to plan for RGNX (and for me). Let’s see what people think the last 3 months hold for us

Regenxbio reported positive three-year follow-up data from its Phase II ALTITUDE trial of surabgene lomparvovec (sura-vec) in nonproliferative diabetic retinopathy. At the pivotal Dose Level 3, 60% of participants who reached the three-year milestone achieved more than a two-step improvement on the Diabetic Retinopathy Severity Scale, a clinically meaningful threshold. No vision-threatening events or additional diabetic retinopathy treatments were reported among those participants. The data were presented Sept. 24 at the Retina Society’s 59th Annual Scientific Meeting in Los Angeles. Chief Medical Officer Steve Pakola described the results as encouraging, while the same Dose Level 3 formulation is being evaluated in the ongoing Phase IIb/III NAAVIGATE pivotal trial. The three-year durability results therefore provide additional clinical support for the dose selected for the company’s registrational program. $RGNX

$RGNX I'd realistically expect an ex-US RGX-202 partnership around H2 2027-H1 2028. If a major partner were highly interested at this stage, a deal could arguably have happened already. With the remaining inherent safety and efficacy risk, and the need for a randomized confirmatory trial for broader regulatory validation, including in Europe, partners may prefer to wait until AFFINITY RISE is well underway. But That would significantly strengthen RGNX's negotiating position

$RGNX can anyone tell when they will submit BLA for 302 ? They were suppose to submit in September ?