Nanotransfer

What opportunities are there for cell and gene therapy? This is the last post in our SWOT analysis on the CGT space in the run up to Meeting on the Mesa, and we're ending things on a positive note with O - Opportunities! Check them out below: 𝐓𝐚𝐫𝐠𝐞𝐭𝐞𝐝 𝐃𝐞𝐥𝐢𝐯𝐞𝐫𝐲: ↳ This means developing delivery methods that target tissues and organs beyond the liver. Increased delivery efficacy would also reduce the severity of side-effects (including the dreaded cytokine release syndrome). ↳ Recent developments in this space are Flagship Pioneering's launch of Mirai Bio, and NanoVation Therapeutics™ partnering with Novo Nordisk. 𝐑𝐨𝐛𝐨𝐭𝐢𝐜𝐬 & 𝐀𝐮𝐭𝐨𝐦𝐚𝐭𝐢𝐨𝐧: ↳ Cell and gene therapy manufacturing is still very labour intensive and not overly standardized. Driving innovation here would decrease costs, reduce batch failure risks, and increase accessibility of these therapies. ↳ Companies to watch in this space include Adva Biotechnology, Astraveus, Cellares, Cellular Origins, Lonza (their Cocoon platform), Limula, Multiply Labs, and Ori Biotech. 𝐀𝐮𝐭𝐨𝐢𝐦𝐦𝐮𝐧𝐞 𝐈𝐧𝐝𝐢𝐜𝐚𝐭𝐢𝐨𝐧𝐬: ↳ Cell therapies may offer a safe and effective pathway to treating several chronic autoimmune diseases, including lupus and multiple sclerosis. ↳ Companies to watch in this space include Artiva Biotherapeutics, Kyverna Therapeutics, iCell Gene Therapeutics, Cartesian Therapeutics, Capstan Therapeutics, Cabaletta Bio, Sonoma Biotherapeutics, CRISPR Therapeutics, Caribou Biosciences, Nkarta, Inc., and Takeda. ↳ Here's a more detailed overview if you're curious (featuring the above players): https://bit.ly/4eOhXeN 𝐀𝐥𝐥𝐨𝐠𝐞𝐧𝐞𝐢𝐜/𝐈𝐧-𝐕𝐢𝐯𝐨 𝐂𝐞𝐥𝐥 𝐓𝐡𝐞𝐫𝐚𝐩𝐢𝐞𝐬: ↳ Donor-based therapies would reduce variation that's inherent to autologous cell therapies and would enable mass production of doses - much more efficient than autologous, single patient batches. ↳ Here's a list of allogeneic cell therapy biotechs from our friends at Labiotech.eu: https://lnkd.in/eYeH4azT ↳ For in-vivo cell therapies, check out this article from Nature: https://lnkd.in/ehZwtpXj 𝐒𝐨𝐥𝐢𝐝 𝐓𝐮𝐦𝐨𝐫𝐬: ↳ Majority of currently approved cell therapy products are for liquid tumors, with progress slowly being made towards solid tumor indications. ↳ First such approval was earlier this year with Iovance Biotherapeutics, Inc.'s Amtagvi for advanced melanoma. Turnstone Biologics is another player to watch in this space. I haven't covered all the opportunities here, there's so many to list! (eg. different cell types beyond the T or NK cell, non-viral delivery, cold-chain innovations and delivery logistics, multi-gene associated genetic diseases for gene therapy). Would love to read your favorite CGT opportunity in the comments. Previous posts on: • Strengths (https://bit.ly/3TS8M4K) • Weaknesses (https://bit.ly/3NcO4sE) • Threats (https://bit.ly/4gTCOis)

What's threatening the cell & gene therapy industry? In the run up to Meeting on the Mesa, we're doing a high-level SWOT analysis on the CGT space, one letter a day until Friday. Today we're covering T (Threats) and bucking the SWOT order so we can end Friday on O (Opportunities). Previous posts on Strengths (https://bit.ly/3TS8M4K) and Weaknesses (https://bit.ly/3NcO4sE). 𝐑𝐞𝐠𝐮𝐥𝐚𝐭𝐨𝐫𝐲 𝐇𝐮𝐫𝐝𝐥𝐞𝐬 ↳ Regulators have dealt with small molecules for over a century, and biologics since the mid-80s, but the CGT approval floodgates didn't really open until the middle of the last decade. As such, regulatory guidance may shift more rapidly than with other modalities, meaning biotech companies may face additional hurdles in ensuring their CGTs meet changing submission and approval requirements. 𝐅𝐢𝐫𝐬𝐭-𝐋𝐢𝐧𝐞 𝐓𝐡𝐞𝐫𝐚𝐩𝐢𝐞𝐬 ↳ We're all playing for the same team in that the patient is the ultimate priority; if a better Duchenne's or liquid tumor small molecule or biologic comes along to usurp the CGT wunderkind, we should celebrate as patients ultimately benefit. But if that better therapy is ahead of a CGT in the treatment line, then it's market potential and long-term commercial viability can be severely compromised. ↳ I'm not advocating for CGTs to be artificially prioritized; instead, by proving superior efficacy and safety, certain CGTs will naturally move into the first-line. 𝐃𝐨𝐜𝐭𝐨𝐫 & 𝐏𝐚𝐭𝐢𝐞𝐧𝐭 𝐀𝐝𝐨𝐩𝐭𝐢𝐨𝐧 ↳ From the doctor's perspective, and especially for chronic conditions, the risk of adverse events may prove too costly to consider for their patients, especially if non-CGT alternatives exist. ↳ Patients unfamiliar with CGTs may be deterred by the high out-of-pocket costs (depending on the medical system), extended hospital stays, and conditioning regimens, especially if more affordable and safer alternatives exist. 𝐈𝐧𝐯𝐞𝐬𝐭𝐨𝐫 𝐏𝐞𝐫𝐜𝐞𝐩𝐭𝐢𝐨𝐧 ↳ The numerous examples of approved therapies running into hurdles (Roctavian, Hemgenix commercializations; bluebird bio struggling with three approved CGTs) may make some investors wary of the potential for reliable returns. Given this unpredictability, many investors may lean toward more established modalities with clearer paths to profitability (especially in down economies). ↳ This makes it exceptionally tough for early-stage CGT ventures, who may face even greater difficulty in attracting capital due to these developmental and commercial uncertainties. 𝐏𝐫𝐢𝐜𝐢𝐧𝐠 𝐏𝐫𝐞𝐬𝐬𝐮𝐫𝐞𝐬 ↳ CGTs come with a hefty price tag, which can lead patients and healthcare providers to favor less costly, repeat-dosing therapies. ↳ Payers, including both public and private insurers, may be unwilling to cover CGTs at scale, particularly if cost-effectiveness remains in question compared to conventional therapies. Anything I missed or want to add your 2 cents? Would love to hear from you below!

What weaknesses plague cell & gene therapy? In the run up to Meeting on the Mesa, we're doing a high-level SWOT analysis on the CGT space, one letter a day until Friday. Today we're covering W (weaknesses) - and unlike CGT's one big strength (covered yesterday: https://bit.ly/3TS8M4K), there is an unfortunate myriad of weaknesses within the current CGT space. 1) 𝐇𝐢𝐠𝐡 𝐂𝐨𝐬𝐭𝐬 ↳ Cell & gene therapies are the most expensive therapies by dose on the planet: Hemgenix, CSL Behring's gene therapy answer to hemophilia B, is pegged at $3.5M per dose. For CAR-T cell therapies, estimates range from $500k-$1M per treatment round. ↳ If the CGT product is aimed towards a chronic, rather than imminently deadly, condition, it can sometimes be hard to justify the upfront cost. ↳ Current payer models, especially in the US, are not equipped to manage the long-term costs of CGTs that may be applicable to larger patient populations. 2) 𝐂𝐨𝐦𝐩𝐥𝐞𝐱 𝐌𝐚𝐧𝐮𝐟𝐚𝐜𝐭𝐮𝐫𝐢𝐧𝐠 ↳ Dealing with living (and viral) systems comes at the price of vastly increased complexity, and many processes within the manufacturing chain are still reliant on manual interventions; these manual steps not only increase costs but also introduce variability, making it harder to ensure consistent product quality at scale. Though great strides are being made in automation and robotics (which we'll touch on in "O-Opportunities"), the current manual state of play contributes in-part to the above high prices. ↳ This complexity also impedes their commercialization, especially as we move to more common approved indications within CGT. 3) 𝐒𝐢𝐝𝐞 𝐄𝐟𝐟𝐞𝐜𝐭𝐬 ↳ Viral vectors (e.g., AAV) can cause immune-related adverse events and are usually not redoseable, while allogeneic cell therapies may face graft-versus-host disease. Additionally, CGT conditioning often involves chemotherapy, compounding risk. 4) 𝐋𝐨𝐠𝐢𝐬𝐭𝐢𝐜𝐚𝐥 𝐂𝐡𝐚𝐥𝐥𝐞𝐧𝐠𝐞𝐬 ↳ The SOP for CGT delivery is cryogenically frozen doses, hindering their access to locations without reliable cold chains. ↳ The increased side effect risk mentioned above also means that CGT's need to be administered at hospitals where these side effects can be managed, further hindering access to those in rural areas. 5) 𝐅𝐢𝐧𝐚𝐧𝐜𝐢𝐧𝐠 𝐔𝐧𝐜𝐞𝐫𝐭𝐚𝐢𝐧𝐭𝐢𝐞𝐬 ↳ Though things are slowly improving in biotech investment, CGT investment isn't recovering as rapidly as their small molecule and biologic peers. The stellar development costs, including the complex manufacturing mentioned above, makes CGT a tough sell. To not end the week on a "Threat"ening note, we're skewing the SWOT order and doing Threats tomorrow (so we can end the week on some positive Opportunities). —————————— Get the top biotech and pharma industry news, straight to your inbox and all in one email - that's TLDR Biotech. 🧬🔔

What strengths does the cell & gene industry have? In the run up to Meeting on the Mesa, we're doing a high-level SWOT analysis on the CGT space, one letter a day until Friday. Today we're covering S (strengths) - and the biggest strength within the CGT space, and the big reason so much money, effort, and hope is being poured into this space, is this: Cell & gene therapies have 𝐎𝐧𝐞-𝐚𝐧𝐝-𝐃𝐨𝐧𝐞 𝐂𝐮𝐫𝐚𝐭𝐢𝐯𝐞 𝐏𝐨𝐭𝐞𝐧𝐭𝐢𝐚𝐥. This means: • Certain previously incurable conditions are now curable. ↳ Eg. Novartis' Zolgensma effectively cures certain pediatric forms of spinal muscular atrophy in patients under 2 years old. • For certain deadly diseases, there are now more, arguably more effective, treatment options for patients. ↳ There are now six separate CAR-T therapies FDA approved to treat different liquid tumors: BMS and 2seventy bio's Abecma, BMS' Breyanzi, J&J & Legend Biotech's Carvykti, Novartis' Kymriah, Kite Pharma's (Gilead) Tecartus and Yescarta. • Certain chronic conditions are now curable, or at least a single treatment can stave off symptoms for years. ↳ This includes CSL Behring's Hemgenix for hemophilia B, bluebird bio's Lyfgenia or CRISPR Therapeutics and Vertex's Casgevy for sickle cell disease, and Casgevy again for transfusion dependent beta thalassemia. Though there is much strength in CGT's one-and-done promise, there are also quite a few weaknesses hindering it's mass appeal - we'll be covering that tomorrow. —————————— Get the top biotech and pharma industry news, straight to your inbox and all in one email - that's TLDR Biotech. 🧬🔔 #biotech #pharma #pharmanews #biotechnews #tldrbiotech

Excited to introduce our 9th Generation! Amplify Dynamics EYWA Biotech Nanotransfer Patagon Fiber / DyeGon Toolkit to Examine Lifelike Language (TELL)

Wow, this Andrew Pannu is good and you should follow his summaries. And here is a landscape of many gene therapy products in different stages - pretty useful. My new favorite right after BioPharmaTrend.com 's target novelty assessment (some aspects of which I disagree but a gargantuan amount of work).

Nanotransfer esta buscando sumar un/a investigador/a Junior para unirse al equipo de I+D en Córdoba, Argentina. Se valorará experiencia previa en: . Manipulación de cultivos bacterianos . Manejo de técnicas de biología celular y molecular básicas (transformación de bacterias, miniprep, preparación de geles de agarosa) . Síntesis y caracterización de nanomateriales. Además, buscamos  una actitud proactiva, capacidad de trabajar en equipo y una excelente comunicación. Sobre Nanotransfer: La terapia génica tiene el potencial de transformar la vida de millones de personas que padecen enfermedades genéticas. Sin embargo, el desafío actual radica en desarrollar sistemas de delivey de genes mas eficientes para recuperar la función de las células dañadas en estos pacientes.   Nanotransfer está aquí para cambiar el juego. Al producir los vehículos adecuados para las terapias génicas, buscamos liberar todo el potencial de estos tratamientos. Trabajamos  para lograr que la próxima generación de estas terapias  puedan llegar a todos los pacientes, democratizando asi el acceso y mejorando exponencialmente la calidad de vida de las personas. ¿Cómo postularte? Envía tu CV a cecilia@nanotransfer.bio   Fecha limite de recepción de CV: 19 de julio 2024 

Gene therapy has the potential to transform the lives of millions suffering from genetic conditions. This innovative technique modifies a person’s genes to treat a disease, addressing root causes at the molecular level and offering hope for lasting cures. However, the challenge in the recent years has been delivering these functional genes effectively into damaged cells. Nanotransfer is here to change the game. By producing the right vehicles for gene therapies, they are unlocking the full potential of these treatments. Their groundbreaking work ensures that next-generation therapies can reach all patients, democratizing access to gene therapies and exponentially improving people's quality of life. The Ganesha Lab welcomes Nanotransfer to its Ninth Generation of #BIGinBIO!