Pediatric Brain Tumor Foundation’s Post

Projecting ourselves, hopefully, to an approval: an interesting nuance on cell therapy (allogeneic but not universal, thus requiring the maintenance of an HLA-typed inventory). And thus, another one with a, hem, *interesting* business model. But given the almost-complete lack of *business* in CGT these days (outside of CAR-Ts), we'll watch anything! #cellandgenetherapy

🌟 Latest News Highlights! 🌟 1. 🧪 Reviva Pharmaceuticals secures #EU patent for its innovative pulmonary hypertension drug! 2. 🤝 XyloCor Therapeutics, Inc. partners with SmartCella Holding AB to advance cardiovascular therapies! 3. 👁️ OKYO Pharma makes strides in developing new therapies for dry eye syndrome! 4. 📑 Elevar Therapeutics resubmits NDA for Rivoceranib, aiming to bring new hope to cancer patients! 💬 Join the Conversation! Which news excites you the most? Share your thoughts below! 🗣️ 🔗 Stay tuned for more updates and exclusive content! #PharmaNow #PharmaNews #HealthcareInnovation #Partnerships #MedicalAdvancements #StayTuned

Today Atara announced that it submitted a Biologics License Application (BLA) to the U.S. FDA for tabelecleucel (tab-cel®) indicated as monotherapy for treatment of adult and pediatric patients two years of age and older with Epstein-Barr virus (#EBV) positive post-transplant lymphoproliferative disease (#PTLD) who have received at least one prior therapy. For solid organ transplant patients, prior therapy includes chemotherapy unless chemotherapy is inappropriate. This represents the first-ever #allogeneic #Tcell therapy BLA submission to the FDA, and if approved, tab-cel would be the first approved therapy in the U.S. for EBV+ PTLD. Read more about this significant milestone for Atara: https://bit.ly/4dL7elE

On May 15th we announced a partnership with Enable Injections to develop a combination product to treat advanced Parkinson's disease. Our chemistry team at Serina spent four years trying to solve the challenges of programming the release of apomorphine from our proprietary POZ polymer. We solved it. We are relentless. SER-252 is a potential breakthrough therapy for advanced disease. We anticipate entering clinical studies in 2025 with a twice per week subcutaneous injection of apomorphine that has the potential to reduce “OFF” time, with no adverse skin reactions and no need for a doctor or nurse to administer it. SER-252 is a preventive therapy (not a rescue therapy) with the potential to increase “ON” time, prevent dyskinesia, and may allow some patients to titrate off all L-DOPA. Leveraging our partnership with Enable Injections, SER-252 is being developed using enFuse®, the innovative wearable drug delivery platform from Enable that allows patient self-administration with rapid treatment time. Go to www.enableinjections.com and www.serinatherapeutics.com to learn more.

Join us at the 18th Annual Pain Therapeutics Summit (https://lnkd.in/evDCNbu) on October 28-29, 2024 in Boston, MA! On the second day, our CEO Prof. Geert Jan Groeneveld will give a presentation on proof-of-concept in early phase clinical studies of new analgesics. CHDR has created a comprehensive nociceptive test battery, PainCart, to screen new analgesic compounds for their potential to influence various pain mechanisms, both nociceptive and neuropathic. This allows us to determine the analgesic potency and profile of new drugs in healthy volunteers, establishing the active concentration range to aid in dose selection for initial patient studies. PainCart has been used to generate proof-of-concept for Nav1.8 blockers, advancing them to phase 2 and phase 3 trials, and to make early no-go decisions for clinical drug programs. In his presentation, Prof. Groeneveld will introduce PainCart and he will provide examples of both types of studies. #clinicalresearch #pain #painresearch

Sage Therapeutics/Biogen Secure FDA Approval for Zuranolone (ZURZUVAE™) as the First Oral Treatment for Postpartum Depression | https://lnkd.in/g8kj-w-a Following the 2019 approval of brexanolone (ZULRESSO™) for IV treatment of postpartum depression (PPD), in August 2023, the FDA approved zuranolone (ZURZUVAE™), Sage Therapeutics / Biogen's oral positive allosteric modulator of CNS GABA signaling for PPD. Starting from an endogenous neurosteroid scaffold, Sage Therapeutics’ lead optimization campaign led to an clean, orally bioavailable compound which was able to engage GABA receptors in vivo. In the clinic, zuranolone showed efficacy in patients suffering from postpartum depression, but the results were more mixed in trials for patients with major depressive disorder (MDD), a much more valuable indication. Ultimately the Sage/Biogen’s joint PDD/MDD NDA resulted in approval for PPD, but the issuing of a CRL for use in patients with MDD. check out the full article | https://lnkd.in/g8kj-w-a

OKYO Pharma Ltd (NASDAQ:OKYO) CEO Gary Jacob joined Proactive to discuss recent developments in the company’s Phase 2 clinical trial for OK-101, an investigational therapy targeting neuropathic corneal pain (NCP). Jacob highlighted a significant milestone for the trial: the first patient dosed. The CEO also noted that OK-101 has previously shown promising results in safety and efficacy during earlier trials for dry eye disease. Proactive: You've got some news out about your Phase 2 trial for OK-101. Why don't we start there? Let's remind everyone a little bit about this particular drug and what it's designed to potentially do. Gary Jacob: Well, we're very... http://ow.ly/PZYU105N2Wu

A massive congratulations to Atara Biotherapeutics, where I worked for almost 3 years, a pioneering allogeneic (aka off-the-shelf) Cell Therapy company. Why is this an important development? The first cell therapies were autologous, meaning each therapy was derived from the patient and used for the same patient. In contrast, allogeneic cell therapies are off-the-shelf. This distinction is crucial for three reasons: 1) Cost 🤑 : having a medicine that is off-the-shelf is substantially cheaper than having to make it personalized. 2) Speed 👟 : whereas in autologous therapy the patient had to donate cells, and having to wait to get them back, off-the-shelf means that the medicine is ready for the patient. 3) Access ♿ : sometimes those patients are so sick that the cells are not usable anymore, by having off-the-shelf EVERYONE has access to it. Amazing congrats to all the hard work! I still remembering going to conferences representing Atara where I was the odd-man out because I was the only person in the room asking questions from the allogeneic perspective. #CellTherapy #Biotech #Cancer #Tcells #Atara

#Radiopharmaceutical clinical trials face a host of logistical challenges, often leading to delays. Learn how we leveraged proven #RLT expertise to help our sponsor meet or beat all critical milestones, securing #FDA approval in under four years. https://bit.ly/3Z4vydo #clinicaltrials #Radioligand #isotopes #clinicalresearch #psicro #radiopharmaceuticals #theranostic #diagnostic #imagingagents #oncology

In May, US HCPs discussed trial results presented at the American Thoracic Society's International Conference. Here are some key points: • HCPs shared research showing that beta blocker bisoprolol is a safe treatment option for cardiac disease in #COPD patients. • They celebrated the positive NOTUS trial results involving Sanofi-Regeneron’s monoclonal antibody Dupixent (dupilumab) and its subsequent priority FDA review. They celebrated it as a “big win for patient care” and considered it among the new biologics for COPD treatment and research. • Following the FDA approval of Verona Pharma's Ohtuvayre (ensifentrine) on 26th June as maintenance treatment for COPD, US HCPs reposted about the “exciting news”, considering it as “potent” and celebrating it as the first in its class to be approved by the FDA for COPD in many years. 👉 Read the full COPD tracker here to find out more! https://lnkd.in/eU9T2QnH To stay up-to-date with our monthly trackers, sign up to CREATION Knowledge https://lnkd.in/e7NbQFQA