Alexander Coppen

👨‍🔬💬 𝐂𝐞𝐥𝐥 & 𝐆𝐞𝐧𝐞 𝐓𝐡𝐞𝐫𝐚𝐩𝐲 𝐏𝐨𝐬𝐢𝐭𝐢𝐯𝐢𝐭𝐲: 𝟐𝟔𝐭𝐡 𝐒𝐞𝐩𝐭𝐞𝐦𝐛𝐞𝐫 𝟐𝟎𝟐𝟓 👩‍🔬💬 🧬 uniQure announced positive topline results from its pivotal Phase I/II study of AMT-130, a gene therapy for Huntington’s disease. High-dose AMT-130 significantly slowed disease progression over 36 months, meeting the primary endpoint and a key secondary endpoint compared to an external control. Favorable trends were observed across cognitive and motor measures, with reduced CSF NfL levels suggesting decreased neurodegeneration. 🧬 Sanofi’s SAR446268, a one-time AAV gene therapy for non-congenital myotonic dystrophy type 1 (DM1), has received FDA Fast Track designation to accelerate its development and review. The therapy uses RNA interference to silence DMPK expression, aiming to remove toxic RNA foci, restore normal splicing, and improve muscle function. It targets key DM1 symptoms, including progressive muscle weakness and myotonia, and addresses systemic effects on heart, lungs, and endocrine function. 🧬 Cure Rare Disease announced that the FDA has granted Orphan Drug Designation for its gene therapy CRD-003 to treat Limb-Girdle Muscular Dystrophy Type R9 (LGMD2i/R9), which currently has no approved treatments. CRD-003 uses an AAV vector to deliver FKRP under a muscle-specific promoter, restoring alpha-dystroglycan glycosylation to maintain muscle integrity. The therapy targets progressive skeletal, cardiac, and CNS muscle weakness caused by FKRP mutations. 🦠 Aspen Neuroscience, Inc. initiated Cohort 3 of its ASPIRO Phase 1/2a trial for ANPD001, a personalized autologous dopaminergic neuronal precursor cell therapy for moderate-to-advanced Parkinson’s disease. This cohort uses Aspen’s commercial formulation, enabling scalable, cryopreserved dosing without immunosuppression. ANPD001 is derived from patient skin cells, reprogrammed into iPSCs and differentiated into dopaminergic cells, delivered via a precision MRI-guided system. 🧬 Intellia Therapeutics, Inc. reported positive longer-term Phase 1 data for nexiguran ziclumeran (nex-z) in hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN). A one-time dose achieved deep, durable TTR reductions across patients, including those who had progressed on patisiran. Most patients showed stability or clinically meaningful improvements in neuropathy scores, quality of life, and disability measures. 🦠 Hemostemix Inc. (TSXV: HEM | OTCQB: HMTXF | FSE: 2VF0) has filed FDA Pre-IND 1517 to advance ACP-01 (VesCell™), its autologous stem cell therapy, via a basket Phase I trial across multiple ischemic and vascular conditions. The basket protocol aims to evaluate safety, feasibility, and early efficacy across indications under a unified design, accelerating development and expanding therapeutic potential. 🛎 My aim is to be the messenger when it comes to your CGT news. Follow #mCGT to stay updated! #celltherapy #genetherapy #ATMP #advancedtherapy

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🧠🚨 CNS News 🚨🧠 AbbVie has just made another big move in neuroscience, agreeing to acquire Gilgamesh Pharmaceuticals bretisilocin program in a deal worth up to $1.2 billion. Rather than a full acquisition, AbbVie will take over bretisilocin (GM-2505), while Gilgamesh spins out its other assets into a new entity. Bretisilocin recently hit a 94% remission rate in a Phase 2 trial for major depressive disorder, with patients dosed just twice, two weeks apart. It acts on the 5-HT2A receptor like LSD/psilocybin, but with a shorter half-life, potentially making it more practical for in-clinic use, similar to the model J&J established with Spravato. Could shorter acting psychedelic therapies like bretisilocin be the breakthrough psychiatry has been waiting for? #Psychedelics #CNS #Neuroscience #Biotechnology

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🚀 New Partnership Alert Arkivum has teamed up with PHARMASEAL to deliver a complete end-to-end clinical trial technology solution. By combining Arkivum’s GxP-validated digital archiving with PHARMASEAL’s Engility CTMS & eTMF, sponsors can now: ✅ Build data-retention planning in from day one ✅ Ensure long-term integrity, accessibility & inspection readiness ✅ Stay fully compliant with ICH E6 (R3), EU CTR, and evolving regulations “This partnership will increase awareness of the importance of a fit-for-purpose solution to preserve all essential clinical trial records and data,” said Chris Sigley, CEO of Arkivum. Ricky Lakhani, Chief Product Officer at PHARMASEAL, added, “It guarantees that critical trial records remain accessible, compliant, and usable for decades to come.” This collaboration future-proofs clinical trial records, keeping essential data secure and usable for decades. #ClinicalTrials #LifeScienceNews #BioPharma #CRO

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🚀 Big pharma money is on the move and small biotechs should be paying attention 🚀 Sanofi Ventures just raised $625M to fuel innovation in rare disease, neurology, immunology, and vaccines. For early-stage biotechs, this could mean survival and speed in a tough funding climate. It also means small companies will need sharper market access and pricing strategies earlier than ever. 👉 Will pharma VCs reshape the biotech funding landscape in 2026?

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Why choose a Biotech for your next Pharma Role? 💊 If you're contemplating your next step in pharma, it's time to take a serious look at biotech. The industry is evolving rapidly, and biotech is at the forefront of innovation, agility, and impact. 🔬 Innovation at Speed Biotech companies are pioneering breakthroughs in gene therapy, personalized medicine, and immuno-oncology. Working in biotech means being part of cutting-edge science that’s changing lives, often faster than traditional pharma pipelines allow. 🌍 Purpose-Driven Work Smaller teams, flatter structures, and mission-focused cultures mean your work directly contributes to transformative outcomes. You’re not just a cog in the machine, you’re a catalyst for change. ⏩ Career Acceleration Biotech environments offer exposure to cross-functional roles, strategic decision-making, and leadership opportunities early in your career. If you're looking to grow fast and stretch your capabilities, biotech is the place to be. 🤝 Collaboration & Culture With a strong emphasis on collaboration, transparency, and innovation, biotech firms often foster a dynamic and inclusive culture that values every voice. Whether you're a scientist, statistician, or operator, the biotech sector offers a unique blend of purpose and possibility. The future of medicine is being built here, why not be part of it? #biotech #careermove

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