Cara Wykowski

Looking forward to Dr. Dan DeAngelo’s comprehensive review of the pivotal, Phase 3 results investigating standard salvage CT +/- Uproleselan in relapsed and refractory AML. This is a large, multi-national, placebo-controlled dataset (N=388) that may help address many important questions in the management of R/R AML.

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Proud to have fellow Biotechnology Innovation Organization board member and Arcutis Biotherapeutics, Inc. (Nasdaq: ARQT) CEO Frank Watanabe testify at the House Ways and Means Committee hearing on July 12th in Salt Lake City. Frank highlighted the contributions the biotech industry has made to human health and the economy: "In the last quarter century, American life expectancy has continued its steady increase upwards, with roughly one-third of that improvement directly attributable to biopharmaceutical innovations.” The big picture: The industry’s direct and indirect contribution to the U.S. GDP is anywhere from 2-7%, with an economic impact of $2.9 trillion in 2021. Biotech capabilities enhance national security by ensuring we can care for ourselves. Legislative proposals that would support innovation include: - The Ensuring Pathways to Innovative Cures (EPIC) Act, which would give small molecule drugs the same 13-year protection from price controls as biologics, and the ORPHAN Cures Act, which would exempt orphan drugs from price controls even if approved for more than one indication. - The Tax Relief for American Families and Workers Act (H.R. 7024), which was passed by the House earlier this year, to restore the R&D tax deduction. Allow for monetization of net operating losses (NOLs) and reform Section 382 on NOLs. - Improve the capital gains exemption under Section 1202 to encourage investment in small biotechs. - Ensure strong strong patent protections and reasonable regulation of M&A activity. Proud of the work BIO does to support the industry, and proud to be a part of community that works tirelessly to drive innovation and improve human and planetary health. #patientsfirst #biotechnology #innovation #humanhealth #sustainability

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Great news that Gilead’s PURPOSE 1 trial has demonstrated 100% efficacy of Lenacapavir, a twice-yearly injectable, in preventing new HIV infections among cisgender women and adolescent girls. These results are especially significant given adolescent girls and young women are particularly vulnerable to HIV. Gilead expects results in late 2024/early 2025 from the program’s other pivotal trial which is assessing twice-yearly Lenacapavir for PrEP among cisgender men who have sex with men, transgender men, transgender women and gender non-binary individuals who have sex with partners assigned male at birth in Argentina, Brazil, Mexico, Peru, South Africa, Thailand and the United States - groups who are underrepresented in HIV prevention trials and are disproportionately affected by the HIV epidemic. https://lnkd.in/e7s-d6f4

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The most important female scientist you’ve never heard of… It’s time to tell the story of Dr. Candace Pert—otherwise known as, “The Scarlet Woman of Neuroscience”. Thank you Harini Bhat, PharmD for covering this story on the “Today I Learned in Science” blog. We are huge fans of your work. Link to the full Substack post in bio!  #womeninstem #moleculesofemotion #mindbodyconnection #integrativehealth #holistichealth #functionalmedicine #inspiringwomen #candacepert #drcandacepert #mentalhealthawareness

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Interested in the latest developments at the FDA's Office of Therapeutic Products (#OTP)? Join a free, public webinar on June 14th featuring Dr. Nicole Verdun, OTP's Super Office Director, and Rachael Anatol, Ph.D., Deputy Super Office Director. What you'll learn: The new "Super Office" structure of the OTP Progress on hiring new staff An overview of the OTP's organization Brought to you by Alliance for Regenerative Medicine. Please use the link in the post below. #celltherapy #genetherapy #cellandgenetherapy #advancedtherapies #lifescience #lifesciences #regulatory #structure #biotech #biotechnology #webinar #productupdate #regulatorystructure

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Monica Ravindra Shah, MD, FACC, CTI Clinical Trial and Consulting Services's Chief Medical Officer, had the opportunity to host a forum at Alliance for Regenerative Medicine Cell and Gene Meeting on the Mesa. A few key takeaways for cell and gene therapies in cardiovascular disease (CVD): -Despite the global burden of CVD only a small fraction of advanced therapies are in CV indications -It is important to target patients at the optimal timing of disease progression -A clear understanding of the question you are trying to answer is a critical component of the clinical development plan -There is an opportunity for novel endpoints including biomarkers and intermediate endpoints CTI Clinical Trial and Consulting Services has a team of experts that can help navigate each of these points.

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In this episode, Dr. Lyle Burgoon discusses a study published in JAMA Network Open that claims a link between #prenatal exposure to certain #chemicals and metabolic syndrome risk in children. He analyzes the study using the seven tips he discussed in a previous episode and highlights several flaws and limitations in the study design. He emphasizes the importance of being skeptical and checking biases when evaluating scientific studies. He criticizes the media, particularly CNN, for sensationalizing and misrepresenting the study’s findings, causing unnecessary #fear and confusion among the public. #WednesdayWisdom https://lnkd.in/gKU-FuAD

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The EveryLife Foundation submitted comments to Representatives DeGette and Bucshon, emphasizing the importance of continued advancements under the #21stCenturyCuresAct. As we approach the 10-year anniversary of this landmark legislation, we reflected on the progress made and highlighted additional policy opportunities to solve for the significant unmet need within the rare disease community. Categories included: ☑ Requiring transparent reporting on current existing regulatory authorities ☑Ensure patient experience data is fully reflected as part of FDA’s benefit-risk regulatory framework ☑Build on the significant progress made by the #PFDD movement ☑Establish a Mechanism for Pre-Competitive Science-Focused Drug Development Conversations ☑Establish an FDA Rare Diseases and Conditions Advisory Committee ☑Enhance FDA’s Focus on Very Small Population Challenges ☑Ensure Policy Adequately Supports the Translation of Scientific Advances to Novel Therapies ☑Opportunities for earlier payer engagement ☑Uphold the FDA’s statutory authority by forbidding public and private payers from making coverage policies based on the pathway that was used to obtain FDA approval. ☑Facilitate Access to Innovative Therapies by Removing Barriers to Value and Outcomes Based Payment Arrangements ☑Enhance Access to Timely Diagnostics Read full comments: https://bit.ly/4dEHtCA

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Recent Funding: Nura Bio (SF) raised $68 million in Series A financing to advance its work on treatments for #neurodegenerative diseases. Nura Bio plans to advance its lead drug candidate targeting SARM1, an enzyme involved in nerve cell degeneration in neurodegenerative diseases. The funding will accelerate development toward clinical trials for conditions like #ALS and multiple sclerosis. The round was led by new investor Sanofi Ventures and included contributions from existing investors The Column Group, Samsara BioCapital, and Euclidean Capital Management. Shilpa Sambashivan, a cofounder and its chief scientist, will take over as its CEO and lead efforts to push its first drug program deeper into clinical testing. M&A, Deals, Partnerships: Roivant Sciences has struck another deal to sell one of its “vants.” This time around, the #dermatology focused Dermavant Sciences (Long Beach, CA) will head to Organon in a deal valued up to $1.2 billion. The sale of Dermavant clears the way for Roivant to focus on #autoimmune-focused Immunovant and a slate of upcoming pivotal trials. Organon will be the new owner of Vtama, a non-steroidal topical therapy for plaque #psoriasis that was approved in 2022. The cream is also currently under FDA review for atopic #dermatitis, with a decision expected before the end of the year. Novartis plans to invest over 40 million to establish a #radioligland therapy manufacturing facility in Carlsbad, CA. This new 10,000-square-foot site is part of a larger $200 million effort to bolster the company’s #radiotherapy infrastructure and is expected to create more than 30 local jobs. Radioligand therapy targets #cancer cells while minimizing damage to healthy tissue. This facility will be Novartis' first U.S. manufacturing site in California. FDA Approvals: Novartis' cancer drug Kisqali has received FDA approval for use in patients with HR+/HER2- early-stage breast cancer at high risk of recurrence. The approval follows results from the NATALEE trial, where Kisqali, combined with #endocrine therapy, reduced the risk of #cancer returning by 25%. The drug is already approved for metastatic breast cancer, and this new approval marks a significant development in early breast cancer treatment strategies. Other Interesting News: Federal Reserve lowered interest rates in the biotech sector for the first time in more than four years. While the Fed’s target, at 4.75% to 5%, remains near 15-year highs, the move could spur investment in #biotech companies, which are typically seen as the kind of risky bet investors tend to disfavor when interest rates are elevated. Still, the Fed’s new rate trajectory isn’t a cure-all. Rates remain much higher than they were when biotech markets peaked early in the pandemic. The industry is still feeling the after-effects of a record number of IPOs in 2020 and 2021. Investors have been more selective since, leading to a slower-than-average IPO pace over the past three years.

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The Effects of Cellular Senescence and Senolytics on Healthspan with Dr. James Kirkland Do you know what #senescent (zombie) cells are and how cellular senescence and #senolytics impact health? In this episode of #LongevitybyDesign, me and Ashley Reaver, MS, RD, CSSD are joined by Dr. James Kirkland from the Mayo Clinic, one of the world's leading figures in aging research. This episode is a deep dive into #aging research, cellular senescence, and senolytics. Dr. Kirkland discusses the impact of cellular aging (senescence) on #healthspan and his discovery of the first #senolytic agents that remove senescent cells and extend healthspan, illuminating his pioneering work in aging research. InsideTracker https://lnkd.in/eVw8QU_Q

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David H. Crean and John Foley are thrilled to announce the creation of a new CEO Life Science peer-to-peer advisory group for San Diego CEOs with the leadership team at REF(Renaissance Executive Forum) San Diego. The focus of the REF group will be on more mature companies post-Series A funded companies including those nearing IPO’s and publicly traded companies. The new cohort is expected to begin in early 2025.   REF is a global organization that brings together business leaders in peer advisory groups to enhance their leadership skills and business strategies. Founded in the USA over 30 years ago, REF operates by assembling groups of 12 to 15 business leaders. These groups meet monthly, guided by a Forum Leader, to discuss strategic decisions and personal goals in a confidential environment. REF emphasizes the concept of "Collective Intelligence," where the diverse experiences and backgrounds of members are leveraged to foster better decision-making and personal growth. The organization serves as a hub for over 2,500 entrepreneurs, members, forum leaders, partners, ambassadors, and global speakers, providing a platform for sharing best practices and accessing new opportunities. This peer advisory model is considered more effective than traditional one-on-one coaching, as it builds lasting relationships and provides diverse perspectives that are crucial for both personal and professional development.   The partnership with REF is a recognition of the great work achieved by CEO360 with early-stage life sciences companies over the last 2.5 years plus a desire to take this platform to another level with a larger platform that REF offers for later stage companies. REF currently has a growing international footprint with an unmet need in Life Sciences well beyond the San Diego ecosystem. REF is well positioned to address those needs in 2025 and beyond.   CEO360 will stick to its core and not move away from its early-stage focus. The new REF cohort will be held in addition to the three CEO360 cohorts currently underway in San Diego, the Bay area, and via a new virtual group that will likely involve international leadership. The vision is to create a feeder program where qualified, seed-stage Life Sciences companies can benefit from the power of peer-to-peer advisory as they progress through the stages of funding - Seed, Series A, Series B, and all the way through IPO.   From a timing perspective, the new REF Life Sciences group has just started recruiting CEOs and Ambassadors in the sectors of therapeutics, pharmaceuticals, diagnostics, nutraceuticals, medical devices, and health tech.    Please stay tuned for updates as we embark on this new exciting journey and extend our mentoring support to later-stage Life Sciences CEOs. Below is a link to the REF website.    https://lnkd.in/gcgcgsBy   #Leadership #LifeSciences #CEOs #REF

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Struggling with Treatment Resistant Depression? Discover the groundbreaking potential of ketamine therapy in alleviating depressive symptoms that conventional treatments can't touch. #KetamineTherapy #DepressionTreatment #Stella https://lnkd.in/gPVHWxA4

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Fast track for FDA by June 30 will release its verdict on #Rocket Pharmaceuticals’ BLA for #Kresladi (marnetegragene autotemcel), an investigational #gene therapy the New Jersey-based biotech is proposing as a treatment for severe #leukocyte adhesion deficiency-1 (LAD-1). Severe LAD-1 is a rare #pediatric disorder caused by mutations in the #ITGB2 gene, which encodes for a crucial component of the CD18 protein. Under healthy circumstances, #CD18 helps leukocytes exit blood vessels to combat infections. In severe #LAD-1, which typically manifests during early childhood, patients suffer from recurrent and unresponsive bacterial and fungal #infections.

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The road to success is rarely straightforward, but today we celebrate a significant milestone in the fight against Alzheimer's. A #donanemab has shown promising effectiveness, bringing hope to millions of patients and their families. This breakthrough is a testament to the relentless dedication of researchers and medical professionals worldwide. Let's continue to support and drive innovation in medical research, for a future where Alzheimer's is a challenge we have conquered. #AlzheimersResearch #MedicalInnovation #HopeForTheFuture

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