{"id":81252,"date":"2026-06-11T06:17:24","date_gmt":"2026-06-11T06:17:24","guid":{"rendered":"https:\/\/www.europesays.com\/ch\/81252\/"},"modified":"2026-06-11T06:17:24","modified_gmt":"2026-06-11T06:17:24","slug":"novartis-always-looking-for-new-ways-to-shuttle-drugs-to-brain","status":"publish","type":"post","link":"https:\/\/www.europesays.com\/ch\/81252\/","title":{"rendered":"Novartis \u2018always looking\u2019 for new ways to shuttle drugs to brain"},"content":{"rendered":"

Novartis may be launching an array of vehicles to shuttle drugs across the blood-brain barrier, but executives told Fierce that the Swiss pharma is \u201calways looking\u201d for new ways to traverse this final frontier of neuroscience.<\/p>\n

The company’s most recent play was a $165 million upfront deal with SciNeuro Pharmaceuticals<\/a> in January for the worldwide license to an amyloid beta-targeting antibody for Alzheimer’s disease. One of the appeals of SciNeuro’s monoclonal antibody is how it harnesses transferrin receptor\u00a0(TFR) to drive drugs into the brain, according to Nazem Atassi, M.D., global development head of neurology and gene therapy at Novartis.<\/p>\n

\u201cTFR is the most advanced [modality] in the space to get drugs through the blood-brain barrier,\u201d Atassi told Fierce in an interview last week.<\/p>\n

Finding ways to bypass this barrier has been a key goal for neurodegenerative-minded companies for years. The barrier protects the inner surfaces of the blood vessels inside the brain and controls how certain chemical molecules go into and out of the brain.<\/p>\n

Novartis\u2019 fellow Swiss pharma Roche, which has a brain shuttle bispecific 2+1 amyloid-beta-targeting monoclonal antibody\u00a0in phase 3 development, penned a\u00a0shuttle-focused deal<\/a>\u00a0with Manifold Bio late last year to work on direct-to-vivo AI-guided brain shuttles.<\/p>\n

By that point, Novartis had already placed one neuroscience bet on TFR when it handed $200 million<\/a> to Arrowhead Pharmaceuticals to develop its preclinical siRNA program targeting alpha-synuclein in order to treat Parkinson\u2019s and other synucleinopathies.<\/p>\n

\u201cTFR is very widely expressed, although very highly expressed on the brain endothelium,\u201d Bob Baloh, M.D., Ph.D., global head of neuroscience at Novartis Biomedical Research, told Fierce in the same interview.<\/p>\n

\u201cYou can tune TFR, and we’re seeing those molecules get into the clinic across a variety of companies\u2014including ours,\u201d he said.<\/p>\n

The collaborations with Arrowhead and SciNeuro involve attempting to tune TFR to get drugs into the brain and remain in preclinical testing for now. But Novartis\u2019 work to tune TFR to muscle is further ahead thanks to the pharma\u2019s $12 billion buyout<\/a> of Avidity Biosciences in October 2025.<\/p>\n

That deal gave Novartis ownership of Avidity\u2019s pipeline of clinical-stage antibody-oligonucleotide conjugates for muscle dystrophies. They include the Duchenne muscular dystrophy therapy delpacibart zotadirsen (del-zota), which uses an anti-TFR1 antibody to deliver an oligonucleotide to target cells. The therapy was tied to a statistically significant 25% increase in dystrophin production among people amenable to exon 44 skipping and is currently on track to be submitted to regulators<\/a> in the coming weeks.<\/p>\n


\n \"Nazem<\/p>\n

<\/a><\/p>\n

Nazem Atassi Novartis<\/p>\n

Nazem Atassi, M.D., global development head of neurology and gene therapy at Novartis
\n (Novartis)<\/p>\n

Meanwhile, a phase 3 study for delpacibart etedesiran in myotonic dystrophy Type 1 is due to read out in the second half of the year, with a late-stage study of the DUX4-targetting delpacibart braxlosiran for facioscapulohumeral muscular dystrophy currently enrolling, Atassi explained.<\/p>\n

\u201cIf you look across the three programs in clinic that Avidity has, you will see common themes\u2014they’re all muscle diseases, they all use TFR [to] drive the payload to the muscle, and in all of them you see very robust target engagement,\u201d he said.<\/p>\n

Not content with riding Avidity\u2019s pipeline of muscle dystrophy drugs to regulators, Novartis is also hoping to turn the company\u2019s tech to neurodegenerative diseases like Alzheimer\u2019s and Parkinson\u2019s.<\/p>\n

It\u2019s a \u201clittle bit easier\u201d to tune TFR to muscle because correctly-tuned formats can enter these tissues quite effectively, according to Baloh.\u00a0<\/p>\n

\u201cIt’s a little bit harder to tune for the brain, and that’s why I think if you look across the industry, you’ll see that the brain programs are just a little behind,\u201d he added.<\/p>\n

Whether trying to tackle Alzheimer\u2019s by targeting amyloid beta, tau or neuroinflammation, all companies run headfirst into the blood-brain barrier.<\/p>\n

That’s why Novartis has another iron in the fire thanks to a deal last year to give Sironax $175 million in upfront and near-term payments<\/a> in return for the option to license the Massachusetts-based biotech\u2019s blood-brain barrier crossing tech. For now, the pharma is keeping the specific targets of that pact under wraps.<\/p>\n

Baloh sees the recent progress made across the space as \u201cexciting\u201d because it means getting drugs to the brain has become \u201can engineering problem\u201d rather than remaining a pipe dream.<\/p>\n

\u201cThat’s where we obviously see a lot of opportunities, so we’re doing a whole series of deals now,\u201d he told Fierce.<\/p>\n

Despite having bet on a range of options, the Novartis execs aren\u2019t ruling out splashing more cash in the future.<\/p>\n

\u201cWe believe there could be other shuttle systems, and simultaneously we’re going to investigate if those do perform better or differently from what you can do with TFR, because we really believe that the opportunity space is so large that that’s the right way to approach it,\u201d Baloh said.<\/p>\n

\u201cWe have really strong teams internally working on the blood-brain barrier\u2014they’re collaborating actually with externals and academics,\u201d he continued.\u00a0<\/p>\n

\u201cI don’t think you will really ever only have one solution to this problem,\u201d Baloh said. \u201cHaving a toolbox that you can then use is probably the best way to approach.\u201d\u00a0<\/p>\n

\u201cWe’re absolutely always looking,\u201d Baloh added when asked whether Novartis is eying up other blood-brain barrier plays.<\/p>\n

Atassi agreed that no good solutions are off the table: \u201cBeyond TFR, we’re looking at all the options of how to get drugs across the blood-brain barrier.\u201d<\/p>\n","protected":false},"excerpt":{"rendered":"Novartis may be launching an array of vehicles to shuttle drugs across the blood-brain barrier, but executives told…\n","protected":false},"author":2,"featured_media":81253,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":"","_share_on_mastodon":"0"},"categories":[127],"tags":[15909,19699,7849,1115,10165,3772,9275,42046,2123,206,15076,42047],"class_list":["post-81252","post","type-post","status-publish","format-standard","has-post-thumbnail","category-novartis","tag-alzheimers-disease","tag-arrowhead-pharmaceuticals","tag-avidity-biosciences","tag-biotech","tag-blood-brain-barrier","tag-deals","tag-fierce-biotech-homepage","tag-neurodegenerative-diseases","tag-neuroscience","tag-novartis","tag-parkinsons-disease","tag-sironax"],"share_on_mastodon":{"url":"https:\/\/pubeurope.com\/@ch\/116730019629463203","error":""},"_links":{"self":[{"href":"https:\/\/www.europesays.com\/ch\/wp-json\/wp\/v2\/posts\/81252","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.europesays.com\/ch\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.europesays.com\/ch\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/ch\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/ch\/wp-json\/wp\/v2\/comments?post=81252"}],"version-history":[{"count":0,"href":"https:\/\/www.europesays.com\/ch\/wp-json\/wp\/v2\/posts\/81252\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/ch\/wp-json\/wp\/v2\/media\/81253"}],"wp:attachment":[{"href":"https:\/\/www.europesays.com\/ch\/wp-json\/wp\/v2\/media?parent=81252"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.europesays.com\/ch\/wp-json\/wp\/v2\/categories?post=81252"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.europesays.com\/ch\/wp-json\/wp\/v2\/tags?post=81252"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}