Jin Li

I had the pleasure of paneling the RAPS Regulatory Intelligence opening plenary alongside some exceptional minds in this field. We provided insights into how Reg Intel integrates as a strategic enabler to the business, and how to communicate the changing regulatory landscape to decision-makers. I spoke on how Reg Intel differs from a traditional compliance function, in that RI involves communicating what’s changing and the “WIIFM” (what’s in it for me). This is what your stakeholders and C-Suite want to know! This is different than the equally important roles of compliance teams, which provide evidence generation for how we are meeting and addressing the regulations (think CAPAs and RCA). We also provided insight into how we link our Regulatory Intelligence efforts to successful advocacy strategies. I weighed in that less mature advocacy models involve a bunch of “one-offs” - disparate communication strategies at conferences, when responding to public consultations, when interacting with your trades, etc. However, centering your advocacy plan around set priorities within your business units and clear internal positions helps enable consistency and a “one voice” message. From here, a clear advocacy plan helps regulators and industry move forward together in better serving the patients! This is the ultimate mission. Thank you Regulatory Affairs Professionals Society (RAPS) for this incredible opportunity! And to my co-panelists (whom I learned a great deal from during this session as well!)

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How might an increase in outpatient utilization of CAR-Ts be impacted by 340B discounts? Read on for our quick take on this evolving market dynamic.

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AEON Biopharma, Inc. announces its FDA BPD Type 2a meeting for ABP-450 - a potential BOTOX biosimilar - scheduled for November 19. The meeting will review its analytical development plan and early similarity data, marking a key step in advancing ABP-450 through the biosimilar pathway. 💉

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🧬 𝗥𝗮𝗿𝗲 𝗗𝗶𝘀𝗲𝗮𝘀𝗲 𝗪𝗲𝗲𝗸𝗹𝘆 - 𝗞𝗲𝘆 𝗠𝗼𝘃𝗲𝘀 & 𝗠𝗶𝗹𝗲𝘀𝘁𝗼𝗻𝗲𝘀 - 𝟭𝟱𝘁𝗵 𝗢𝗰𝘁𝗼𝗯𝗲𝗿 𝗥𝗲𝗴𝘂𝗹𝗮𝘁𝗼𝗿𝘆 & 𝗙𝗗𝗔 Denali Therapeutics (MPS II / Hunter): FDA extended the 𝘁𝗶𝘃𝗶𝗱𝗲𝗻𝗼𝗳𝘂𝘀𝗽 alfa BLA review to April 5, 2026 (a major amendment, not related to safety or efficacy). Rocket Pharmaceuticals (LAD-I): FDA accepted the BLA resubmission for 𝗞𝗿𝗲𝘀𝗹𝗮𝗱𝗶; PDUFA March 28, 2026. Abeona Therapeutics (XLRS): ABO-503 selected for the FDA’s Rare Disease Endpoint Advancement (RDEA) pilot - enabling earlier alignment on meaningful clinical endpoints in small populations. ReviR Therapeutics (CMT): Granted Orphan Drug Designation for 𝗥𝗧𝗫-𝟭𝟭𝟳, a first-in-class small molecule therapy. Aardvark Therapeutics (PWS): FDA alignment on Phase 3 HERO protocol expansion to include patients aged 10+. 𝗙𝗶𝗿𝘀𝘁-𝗶𝗻-𝗮-𝗱𝗲𝗰𝗮𝗱𝗲 𝗮𝗽𝗽𝗿𝗼𝘃𝗮𝗹 Boehringer Ingelheim’s 𝗝𝗮𝘀𝗰𝗮𝘆𝗱 (nerandomilast) for IPF: The first new adult treatment for idiopathic pulmonary fibrosis in over 10 years - a major win for patients living with this progressive lung disease. 𝗖𝗹𝗶𝗻𝗶𝗰𝗮𝗹 𝗽𝗿𝗼𝗴𝗿𝗲𝘀𝘀 4D Molecular Therapeutics (CF): 𝟰𝗗-𝟳𝟭𝟬 moves into Phase 2, supported by up to $11M in additional Cystic Fibrosis Foundation funding. Avidity Biosciences, Inc. (𝗗𝗠𝗗-𝟰𝟰): Positive pre-BLA meeting with the FDA; targeting Q1 2026 submission for 𝗱𝗲𝗹-𝘇𝗼𝘁𝗮. Taysha Gene Therapies (Rett): Presented new REVEAL data showing consistent safety and functional improvements across cohorts. Genethon (DMD): Two-year efficacy sustained with its low-dose micro-dystrophin gene therapy (𝗚𝗡𝗧𝟬𝟬𝟬𝟰). CRISPR Therapeutics (AATD): Promising in-vivo gene correction preclinical data for 𝗖𝗧𝗫𝟰𝟲𝟬; first-in-human trial planned for mid-2026. Regeneron (OTOF hearing loss): 𝗖𝗛𝗢𝗥𝗗 Phase I/II data show meaningful hearing gains in most participants; FDA filing preparation underway. 𝗗𝗲𝗮𝗹𝘀 & 𝗳𝘂𝗻𝗱𝗶𝗻𝗴 AviadoBio x UgeneX Therapeutics Co., Ltd.: Up to $413M licensing pact for RP gene therapy 𝗨𝗚𝗫-𝟮𝟬𝟮. Palvella Therapeutics (MLM): Received year-two Orphan Products Grant funding to support the Phase 3 SELVA trial of 𝗤𝗧𝗢𝗥𝗜𝗡 𝗿𝗮𝗽𝗮𝗺𝘆𝗰𝗶𝗻. BioCryst Pharmaceuticals, Inc. to acquire Astria Therapeutics, Inc.: Strengthens its HAE portfolio, adding a long-acting preventative candidate. Johnson & Johnson ↔ Protagonist Therapeutics: J&J is reportedly in talks to acquire Protagonist Therapeutics, potentially deepening their immunology pipeline. insitro & Bristol Myers Squibb Myers: Extended their ALS research collaboration, leveraging Insitro’s AI platform (ChemML) to advance novel ALS targets. Zenas BioPharma: secure a deal for an MS candidate that Biogen had returned to its original partner, InnoCare Pharma. 👀 𝘞𝘩𝘢𝘵 𝘤𝘢𝘶𝘨𝘩𝘵 𝘺𝘰𝘶𝘳 𝘦𝘺𝘦 𝘵𝘩𝘪𝘴 𝘸𝘦𝘦𝘬? Endpoints News | BioSpace | ARTO #raredisease #medicalaffairs #drugdevelopment

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