Spencer Knight’s Post

𝐂𝐆𝐓𝐬 𝐚𝐫𝐞 𝐚𝐜𝐭𝐢𝐯𝐞𝐥𝐲 𝐫𝐞𝐬𝐡𝐚𝐩𝐢𝐧𝐠 𝐡𝐞𝐚𝐥𝐭𝐡𝐜𝐚𝐫𝐞: Clinical trials are evolving beyond oncology 👇 McKesson's fantastic CGT report highlights both the challenges and diversified excitement of the future of cell and gene therapy. The clinical trial data below reveals a robust, established pipeline of innovative therapies across various high-need areas, including: - Oncology: 62 unique cell therapies and 49 gene therapies. - CNS: 44 cell therapies and 41 gene therapies. - Cardiovascular Diseases: 31 cell therapies. ➡️  This certainly highlights the significant unmet medical needs and complexities of various diseases. The diversity of conditions targeted by CGTs demonstrates their potential to treat previously untreatable conditions, paving the way for cures and effective management across multiple therapeutic areas. A long way to go, however, as we continue to advance in this field, the future looks promising for patients who don't have truly curative treatments. 𝐖𝐡𝐚𝐭 𝐞𝐱𝐜𝐢𝐭𝐞𝐬 𝐲𝐨𝐮 𝐦𝐨𝐬𝐭 𝐚𝐛𝐨𝐮𝐭 𝐭𝐡𝐞 𝐟𝐮𝐭𝐮𝐫𝐞 𝐨𝐟 𝐂𝐆𝐓𝐬? (𝘚𝘰𝘶𝘳𝘤𝘦: 𝘔𝘤𝘒𝘦𝘴𝘴𝘰𝘯 𝘢𝘯𝘥 𝘵𝘩𝘦𝘪𝘳 𝘦𝘹𝘤𝘦𝘱𝘵𝘪𝘰𝘯𝘢𝘭 𝘳𝘦𝘱𝘰𝘳𝘵). #celltherapy #genetherapy #biotech #CGTweekly

After given only 2 weeks to live after 4 rounds of chemotherapy for B cell lymphoma, 58 year old Lisa Noble is now cancer free after CAR-T cell therapy. This groundbreaking treatment has emerged and NOW is a proven and transformative option for patients who have exhausted traditional therapies. ➡️ Thanks to CAR-T and the reprogramming of Lisa's immune cells to better recognise and attack cancer cells, she is joyfully celebrating her 25th wedding anniversary and welcoming three new grandchildren into her life. 👉 In addition, the NHS here in England and the University of Cambridge are set to partner and are taking significant steps to expand access to these life-saving therapies. The NHS will open a new cell therapy laboratory in 2025-2026, which will allow for in-house modifications of T cells. Amazing to see cell therapy being provided further access to patients. #celltherapy #oncology #CGTweekly #cellandgenetherapy

𝐊𝐞𝐲 𝐈𝐧𝐬𝐢𝐠𝐡𝐭𝐬 𝐟𝐫𝐨𝐦 𝐭𝐡𝐞 𝐌𝐞𝐬𝐚: 5 Lessons from the CGT Conference 👇 Had a great time in Phoenix last week. 𝐇𝐞𝐫𝐞'𝐬 𝐰𝐡𝐚𝐭 𝐈 𝐥𝐞𝐚𝐫𝐧𝐞𝐝 𝐟𝐫𝐨𝐦 𝐭𝐡𝐞 𝐚𝐧𝐧𝐮𝐚𝐥 𝐀𝐑𝐌 𝐦𝐞𝐞𝐭𝐢𝐧𝐠: 1. Certain CGTs, especially for severe conditions like cancer, require extended inpatient monitoring due to potential severe side effects, such as CRS in CAR-T therapies. 👉 𝐋𝐞𝐬𝐬𝐨𝐧: As more CGTs are approved, the demand for specialised hospital beds will increase. Healthcare providers must scale to ensure safe / effective patient monitoring and treatment. There are not enough treatment centres 2. Clinical data (POC data minimum) is crucial for securing funding from both private investors for cell and gene therapies as well as for an IPO. 3. COGS and commercial viability are closely intertwined and are VITAL in CGTs. 👉 𝐋𝐞𝐬𝐬𝐨𝐧:  By managing production costs, addressing manufacturing earlier on in the drug development process, CGT companies can enhance their sustainability and attract necessary investment for long-term success. 4. Despite tough funding conditions, CGTs are pushing forward, with 14 potential approvals in 2024. Collaboration deals and licensing agreements are keeping the sector alive financially. M&A is lower, large pharma controls CGT. 5. For small biotech, initiating early conversations with regulatory bodies, such as the FDA and EMA, plays a crucial role in the development, scientific hurdles, market readiness as well as commercialisation of innovative therapies. 𝐃𝐨 𝐲𝐨𝐮 𝐛𝐞𝐥𝐢𝐞𝐯𝐞 𝐭𝐫𝐞𝐚𝐭𝐦𝐞𝐧𝐭 𝐜𝐞𝐧𝐭𝐫𝐞𝐬 𝐚𝐧𝐝 𝐭𝐡𝐞 𝐧𝐞𝐞𝐝 𝐟𝐨𝐫 𝐬𝐜𝐚𝐥𝐚𝐛𝐥𝐞 𝐦𝐚𝐧𝐮𝐟𝐚𝐜𝐭𝐮𝐫𝐢𝐧𝐠 𝐰𝐢𝐥𝐥 𝐛𝐞 𝐤𝐞𝐲 𝐟𝐨𝐫 𝐬𝐮𝐜𝐜𝐞𝐬𝐬? #celltherapy #genetherapy #cellandgenetherapy #CGTweekly

𝐋𝐚𝐲𝐨𝐟𝐟𝐬 𝐩𝐞𝐫𝐬𝐢𝐬𝐭 𝐢𝐧 𝐛𝐢𝐨𝐭𝐞𝐜𝐡, It's time to call for change 👇 This week delivered more tough news for biotech as Turnstone Biologics has slashed its workforce by a STAGGERING 60% and Astellas has also downsized its cell therapy team (via Fierce Biotech). These developments highlight the painful challenges our industry is grappling with and emphasise the urgent need for a more strategic adaptation. I can’t help but wonder... It seems the priority is to cut costs and focus solely on lead pipeline candidates. 𝐁𝐮𝐭 𝐢𝐬 𝐭𝐡𝐢𝐬 𝐚 𝐬𝐮𝐬𝐭𝐚𝐢𝐧𝐚𝐛𝐥𝐞 𝐚𝐩𝐩𝐫𝐨𝐚𝐜𝐡? 𝘙𝘦𝘤𝘦𝘯𝘵 𝘓𝘢𝘺𝘰𝘧𝘧 𝘈𝘯𝘯𝘰𝘶𝘯𝘤𝘦𝘮𝘦𝘯𝘵𝘴 👇 - BMS: 2,200 layoffs to save $1.5 billion by 2025. - Bayer: 1,500 layoffs to save $2.6 billion by 2026. - Pfizer: Undisclosed layoffs to save $5.5 billion by 2027. ➡️ While cost-cutting measures may boost short-term financials, the long-term impact on innovation and employee welfare cannot be ignored. Last year, these biopharma giants achieved profits surpassing $30 billion. Yet, they're pursuing aggressive workforce reductions - choosing profits over people. 𝐖𝐞 𝐦𝐮𝐬𝐭 𝐫𝐞𝐭𝐡𝐢𝐧𝐤 𝐨𝐮𝐫 𝐚𝐩𝐩𝐫𝐨𝐚𝐜𝐡. It’s clear that returning investor and shareholder value and managing the risks of clinical trials are critical, but so too is the importance of investing in the workforce that powers innovation. 👉 The rapid regulatory progress and success we’ve seen in Cell & Gene Therapy is a DIRECT result of the talent and dedication of those within the industry. Without these individuals, future breakthroughs are at risk. 𝐓𝐨 𝐭𝐡𝐨𝐬𝐞 𝐢𝐦𝐩𝐚𝐜𝐭𝐞𝐝 𝐛𝐲 𝐥𝐚𝐲𝐨𝐟𝐟𝐬, 𝐦𝐲 𝐭𝐡𝐨𝐮𝐠𝐡𝐭𝐬 𝐚𝐫𝐞 𝐰𝐢𝐭𝐡 𝐲𝐨𝐮. It’s time for the industry to recognise that a healthy, engaged workforce is not an expendable asset - it’s a vital driver of long-term success. 𝘏𝘰𝘸 𝘥𝘰 𝘺𝘰𝘶 𝘣𝘦𝘭𝘪𝘦𝘷𝘦 𝘭𝘢𝘺𝘰𝘧𝘧𝘴 𝘪𝘯 𝘣𝘪𝘰𝘵𝘦𝘤𝘩 𝘸𝘪𝘭𝘭 𝘪𝘯𝘧𝘭𝘶𝘦𝘯𝘤𝘦 𝘤𝘩𝘢𝘯𝘨𝘦 𝘢𝘯𝘥 𝘪𝘯𝘯𝘰𝘷𝘢𝘵𝘪𝘰𝘯? #biotech #celltherapy #genetherapy #CGTweekly

That’s a wrap for #CGMESA24! What an incredible event with even more incredible people. 👉 Fantastic work from the team at ARM as usual. It’s always inspiring to connect at conferences like these with people both leading and driving positive change for patients and advancing our space in meaningful ways. It was truly a pleasure meeting everyone - safe travels home and here’s to continuing the great work! Stay tuned! I will have a few observations to post from this week’s conference arriving early next week. #celltherapy #genetherapy #CGTweekly

Breakthrough in Autoimmune Care:  Allogeneic CAR T Could Change Everything 👇 I may be catching up to the news, but this is something we can’t overlook. For the first time, donor-derived CAR T-cell therapy has put three patients into remission from severe autoimmune diseases like systemic sclerosis. 👉 In a pivotal trial led by Xu Huji from Naval Medical University in Shanghai, scientists used donor T cells, modified with CRISPR technology, to eliminate the rogue B cells driving these conditions. Gene editing x autoimmune. Nice. The results are nothing short of remarkable - one patient, Mr. Gong, returned to work JUST two weeks after receiving the treatment. Why is this a big deal? This could unlock affordable, scalable treatments for over 80 autoimmune diseases. If this approach succeeds in more trials, it could completely reshape how we treat these diseases and bring lasting relief to millions. What’s next? With long-term studies ahead, the potential is immense. If you work in cell therapy, this is the development to watch closely. Nature study, here: https://lnkd.in/dmJ2v6PC #celltherapy #immunology #cellandgenetherapy #CGTweekly

Fantastic first day in chilly Arizona 🏜️ Ready for Day 2 today - excellent catching up with clients and meeting an amazing amount of new people yesterday. Looking forward to it today at #CGMESA24 - feel free to say hello if you see me around! 👋 #celltherapy #genetherapy #CGTweekly

Day 1 of #CGMESA24 in sunny Arizona! Looking forward to connecting with my network and more. If we don’t have a meeting scheduled then feel free to say hi 👋 #CGTweekly #celltherapy #genetherapy

For years, patients with solid tumours have endured limited options and devastating outcomes, leaving families feeling helpless. 👉 BUT change is here. The FDA's approval of Afamitresgene autoleucel (Tecelra) marks a breakthrough in TCR-T cell therapies, now showing real promise against solid tumours. 𝐓𝐡𝐞 𝐩𝐫𝐨𝐨𝐟 𝐢𝐬 𝐢𝐧 𝐭𝐡𝐞 𝐫𝐞𝐬𝐮𝐥𝐭𝐬: - Over 30 clinical trials of engineered TCR-T cell therapies are in progress. Tecelra achieved a 43% response rate in synovial sarcoma. - Notably, Trials targeting MAGE-A4 have shown up to 50% response rates in ovarian, head & neck, and urothelial cancers. - First of Its Kind: Adaptimmune's Tecelra is the first approved engineering TCR-T therapy for solid tumours. ➡️  The tide is turning. These numbers show real progress, offering hope to patients and families who’ve waited too long for a breakthrough. We're finally making strides in the cell therapy fight against solid tumours. Data. Data. Data. It speaks for itself - never underestimate its power. Do you think the wave of TCR cell therapies is arriving in 2025? Full nature article, here: https://lnkd.in/evMrQkzi #celltherapy #oncology #biotech #CGTweekly

𝐁𝐫𝐞𝐚𝐤𝐭𝐡𝐫𝐨𝐮𝐠𝐡 𝐖𝐞𝐞𝐤 𝐟𝐨𝐫 𝐂𝐞𝐥𝐥 𝐓𝐡𝐞𝐫𝐚𝐩𝐲 𝐅𝐮𝐧𝐝𝐢𝐧𝐠: 400 Million secured with an exciting IPO 👇 👉 Cartherics Pty Ltd, successfully secured over AU$15 million in an oversubscribed funding round. This capital for the Melbourne company will support clinical trials for CTH-401, a cell therapy targeting ovarian cancer. ➡️  Enara Bio, located in Oxford, has closed a $32.5 million Series B financing round. Led by Pfizer Ventures and M Ventures, this funding will advance their TCR-based immunotherapies focused on novel Dark Antigens in solid tumours. 👉 Resolution Therapeutics has raised £63.5 million ($85 million) in Series B financing. This UK cell therapy biotech will use the funding to facilitate the advancement of their macrophage therapy, RTX001, into clinical trials for end-stage liver disease. ➡️  Zephyrm Bioscience is set for a successful IPO to fund Phase 3 trials of their cell therapy ZH901, aimed at treating lung conditions and graft-versus-host disease. They have also raised 258 million Chinese yuan ($37 million) to support their development efforts. Excellent to see investments pouring in the U.K, APAC and even Australia. What do you think the impact of these investments will for cell therapy? #celltherapy #genetherapy #cellandgenetherapy #CGTweekly