Podcasts about bluebird bio

This week we are joined by Jason Foster, CEO of Ori Biotech to talk about the future of CGT and why scalable strategic manufacturing is in a make or break moment, his experience scaling Indivior from 5 to 1,100 people culminating in a £3B exit and the powerful culture behind that growth, the Bluebird Bio acquisition and what it means for CGT investment today, and why he is deeply optimistic about Ori Biotech's automated, closed platform. A must-listen for anyone building, investing in, or scaling the next era of biotech.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world. Bluebird bio has made the decision to go private in order to restructure and focus on long-term growth amidst declining valuations and funding challenges in the biotech industry. This move reflects a strategic shift in response to the current market conditions. Johnson & Johnson continues to face legal battles over talc products, with a pending ruling that could potentially resolve the issue. This ongoing litigation highlights the importance of product safety and regulatory compliance in the pharmaceutical industry. In other news, Viking has secured a supply of an obesity pill candidate, demonstrating a commitment to addressing unmet medical needs in the field of obesity treatment. Roche has also made a significant play in the obesity market with a potential $5.3 billion pact, signaling confidence in the potential of this therapeutic area. The COVID-19 pandemic has had lasting impacts on the life sciences sector, prompting companies to adapt and innovate in response to changing market dynamics. The industry continues to evolve in light of these challenges, driving progress and innovation in healthcare. Overall, these developments underscore the dynamic nature of the pharmaceutical and biotech industries, highlighting the importance of strategic decision-making and adaptability in a rapidly changing landscape.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world.The Epic Act has been reintroduced in Congress to remove the IRA's 'pill penalty', with hopes for positive changes. However, budgetary constraints may make it challenging for the current administration to make significant amendments to the IRA. As money continues to flow into weight loss drugs, physicians are prioritizing efficacy and access. Emalex is moving towards FDA approval after a successful Phase III trial for Tourette Syndrome. AstraZeneca also received positive Phase III results for an oral breast cancer drug. SpringWorks Therapeutics successfully turned a Pfizer 'ghost drug' into a victory by keeping a rare disease program alive with the help of advocacy groups. This success story has led experts to believe that this method could be applied to other rare diseases as well. The company serves as a case study for how to mine biopharma's IP storeroom for rare disease drugs.Meanwhile, weight loss doctors are seeking better GLP-1 medications, but are facing challenges with high prices and supply issues. Trump is threatening big pharma with tariffs unless they reshore manufacturing. Bluebird Bio is facing financial challenges and may go private in a deal valued at $30 million. Compounders are suing the FDA over declaring a shortage of certain drugs, while the FTC is tightening merger rules despite the Trump administration's pro-industry stance.These updates show the ever-evolving landscape of the pharmaceutical and biotech industries. Stay tuned for more important news and developments in the field.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world. Robert F. Kennedy Jr., known for his anti-vaccine views, has been chosen by President Trump to lead the Department of Health and Human Services. Kennedy has expressed plans to overhaul the FDA and reduce the NIH's headcount, raising concerns about the future of healthcare regulation. In other news, Celgene shareholders are suing Bristol-Myers Squibb for allegedly avoiding contingent value right payments related to their acquisition deal, while Eyenovia's stock plummets after dropping their lead program due to lack of efficacy. Bluebird Bio is facing a cash shortage with only two quarters of funding left, prompting executives to seek financing to reach breakeven point by 2025. Overall, the pharmaceutical industry had a tumultuous third quarter, with companies like Gilead and Kite cutting employees and closing sites. The industry is in a state of uncertainty as it navigates through these challenges.

Howie and Harlan discuss health and healthcare issues in the headlines, including a powerful—but dangerous—new gene therapy, racial disparities in excess deaths during the COVID pandemic, and the limited insurance coverage for highly effective new obesity drugs.  Links: The Physician Shortage “Opening the Door Wider to International Medical Graduates—The Significance of a New Tennessee Law” “New Licensure Pathway for Some Internationally Trained Physicians” “Brain-drain and health care delivery in developing countries” “Talk of an Immigrant ‘Invasion' Grows in Republican Ads and Speech” Subspecialty Expertise from AI “Towards Democratization of Subspeciality Medical Expertise” Gene Therapy “7 children developed blood cancer after Bluebird Bio gene therapy for rare neurological disease” National Institute of Neurological Disorders and Stroke: Adrenoleukodystrophy An AI Warning from a Nobel Laureate Nobel Prize: Nobel Prize in Physics “Why the Godfather of A.I. Fears What He's Built” “Unions Give Workers a Voice Over How AI Affects Their Jobs” Conflicts of Interest and the Role of Peer Reviewers “Medical journal peer reviewers are paid millions by industry, study finds” “Does industry funding equal conflict of interest? Often it does, Yale authors claim” COVID, Race, and Excess Deaths “Racial and Ethnic Disparities in Age-Specific All-Cause Mortality During the COVID-19 Pandemic” Insurance Coverage for GLP-1 Drugs KFF: 2024 Employer Health Benefits Survey “The Miracle Weight-Loss Drug Is Also a Major Budgetary Threat” CDC: Adult Obesity Facts Mothers in Medicine “So Visibly a Mother” Learn more about the MBA for Executives program at Yale SOM. Email Howie and Harlan comments or questions.

Howie and Harlan discuss health and healthcare issues in the headlines, including a powerful—but dangerous—new gene therapy, racial disparities in excess deaths during the COVID pandemic, and the limited insurance coverage for highly effective new obesity drugs.  Links: The Physician Shortage “Opening the Door Wider to International Medical Graduates—The Significance of a New Tennessee Law” “New Licensure Pathway for Some Internationally Trained Physicians” “Brain-drain and health care delivery in developing countries” “Talk of an Immigrant ‘Invasion' Grows in Republican Ads and Speech” Subspecialty Expertise from AI “Towards Democratization of Subspeciality Medical Expertise” Gene Therapy “7 children developed blood cancer after Bluebird Bio gene therapy for rare neurological disease” National Institute of Neurological Disorders and Stroke: Adrenoleukodystrophy An AI Warning from a Nobel Laureate Nobel Prize: Nobel Prize in Physics “Why the Godfather of A.I. Fears What He's Built” “Unions Give Workers a Voice Over How AI Affects Their Jobs” Conflicts of Interest and the Role of Peer Reviewers “Medical journal peer reviewers are paid millions by industry, study finds” “Does industry funding equal conflict of interest? Often it does, Yale authors claim” COVID, Race, and Excess Deaths “Racial and Ethnic Disparities in Age-Specific All-Cause Mortality During the COVID-19 Pandemic” Insurance Coverage for GLP-1 Drugs KFF: 2024 Employer Health Benefits Survey “The Miracle Weight-Loss Drug Is Also a Major Budgetary Threat” CDC: Adult Obesity Facts Mothers in Medicine “So Visibly a Mother” Learn more about the MBA for Executives program at Yale SOM. Email Howie and Harlan comments or questions.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.GSK has settled Zantac lawsuits for $2.2 billion, resolving 93% of product liability cases regarding allegations that the heartburn drug could cause cancer. Analysts are now shifting focus to the company's vaccines business. In other news, pediatric patients treated with Bluebird Bio's gene therapy developed blood cancers, Pfizer's phase III prostate cancer trial results may lead to a broader label for Talzenna-Xtandi combo, and Gritstone Bio has filed for bankruptcy in an effort to save its clinical research in cancer and infectious disease.On the marketing front, Hershey is using AI to refine its media strategy for the high-stakes Halloween season, with executives emphasizing the importance of targeting and timing, especially with the holiday falling just before the U.S. elections. United Airlines has successfully brought its Kinective media network to market, with the managing director of strategic partnerships sharing lessons learned and discussing future plans. However, a report from Interbrand indicates that a focus on performance marketing has cost brands trillions due to a lack of investment in long-term strategy. PepsiCo Foods' creative chief discussed in-housing and borderless content strategies, while Mountain Dew refreshed its visual identity to align with a new era of marketing. Duracell's CMO shared lessons on bringing low-interest brands into culture, and Diageo highlighted how AI helps them target audiences and navigate regulatory waters.Stay tuned for more updates on upcoming events, company announcements, and resources on customer engagement, retail trends, and more in the marketing industry.

Join us each week as we do a quick review of three compelling stories from the pharma world — one good, one bad and one ugly. Up this week:   The good —FDA approves BMS schizophrenia drug  The bad — bluebird bio announces layoffs  The ugly —J&J talc subsidiary files for bankruptcy

In this episode, pediatric hematologist-oncologist Dr. Anjulika Chawla sits down with undergraduate Anisa Sharma to discuss her journey combatting sickle cell anemia through both clinical and biotech approaches. While practicing at Brown University, Chawla also leads sickle cell clinical research at bluebird bio, a Cambridge-based biotech developing gene therapies. The company's therapy for sickle cell anemia received FDA approval this past year in December. Chawla addresses her experience shifting her focus from treating patients directly to overseeing medical research at bluebird. She also considers bluebird's future in further improving their therapy's impact.

Last month the FDA approved a new treatment for sickle cell disease, the first medical therapy to use CRISPR gene editing technology. It works by identifying the gene or genes causing the disorder, modifying those genes and then returning them to the patient's body.There are now two gene therapies offered by pharmaceutical companies for sickle cell disease: Casgevy from Vertex Pharmaceuticals and CRISPR Therapeutics, and Lyfgenia from BlueBird Bio. But prices for these one-time treatments are steep: Casgevy costs $2.2 million per patient and Lyfgenia $3.1 million.Both promise a full cure, which would be life-changing for patients with this debilitating condition. Over 100,000 Americans, mostly of African descent, have sickle cell disease.This milestone raises more questions: What will be the next disease that CRISPR can help cure? And is it possible to reduce the costs of gene therapy treatments?Ira talks with Dr. Fyodor Urnov, professor of molecular and cell biology and scientific director of technology and translation at the Innovative Genomics Institute, based at the University of California, Berkeley, about the future of CRISPR-based cures.Transcripts for this segment will be available the week after the show airs on sciencefriday.com. To stay updated on all things science, sign up for Science Friday's newsletters.

For the free course "ChatGPT4 for Medical Writers and Editors," go to learnAMAstyle.com Visit Nascentmc.com/podcast for detailed show notes and links.         Erdafitinib for Urothelial Carcinoma: The FDA has fully approved erdafitinib (Balversa) for advanced or metastatic urothelial carcinoma with FGFR3 genetic alterations in adults. This follows its initial accelerated approval and is based on the phase 3 THOR trial results, showing improved survival rates and manageable side effects. Erdafitinib reduced death risk by 36% and had a lower treatment discontinuation rate compared to chemotherapy.        TTFields in NSCLC: The FDA is reviewing a premarket approval application for tumor treating fields (TTFields) combined with standard therapies for platinum-resistant non–small cell lung cancer. TTFields, first approved in 2011, disrupt cancer cell division and showed enhanced survival in NSCLC patients when combined with immune checkpoint inhibitors or docetaxel, without increasing systemic toxicities. The FDA's decision is expected in the second half of 2024.        AI-Powered Device to Detect Skin Cancer: The FDA has approved the first AI-powered handheld device by DermaSensor for assisting in skin cancer detection. It uses AI-driven spectroscopy for analyzing skin lesions and is based on a study involving over 1000 patients. While not a primary screening tool, it aids in detecting melanoma and other skin cancers, especially in patients over 40, and requires further validation testing.        Casgevy for Transfusion-Dependent Beta Thalassemia: The FDA has approved Casgevy (exa-cel), developed by Vertex Pharmaceuticals and CRISPR Therapeutics, for treating transfusion-dependent beta-thalassemia. This follows its approval for sickle cell disease and marks the first CRISPR gene-editing technology application for this condition. The approval came ahead of the anticipated date and follows Bluebird Bio's 2022 approval for a similar gene therapy.        HyQvia for CIDP: HyQvia, an immune globulin infusion 10%, has been approved by the FDA for chronic inflammatory demyelinating polyneuropathy (CIDP) in adults. Initially approved for primary immunodeficiency, HyQvia is the only product combining immunoglobulin with hyaluronidase, allowing for monthly subcutaneous infusions. The approval is based on its demonstrated efficacy in preventing neuromuscular disability relapse.        Physicians' Understanding of FDA Approval Process: A national survey reveals that many physicians have limited understanding of the FDA's drug and medical device approval processes. Only 41% of the surveyed physicians reported moderate or better comprehension of the drug approval process. Despite believing in the adequacy of FDA standards, there's a call for more rigorous post-marketing studies and enhanced education on FDA processes to avoid misconceptions and inaccurate patient advice.

Guest host Nick Shipley, Bio's policy expert joins Daphne Zohar, Brad Loncar, Mike Yee and Yaron Werber on this week's Biotech Hangout. The group discusses policy affecting biotech, including march-in rights, PBMs, IRA and FTC involvement plus bipartisan initiatives like the Orphan Cures Act. They also discuss the state of the XBI and industry sentiment, Bluebird Bio's setbacks following FDA approval and Pfizer's stock after its 2024 forecast revenue. They cover data from Vertex, Icosavax, Arcellx and Legend, plus several ADC deals including Bristol Meyers Squibb, SystImmune, C4 Therapeutics, Merck, Nona Biosciences and Pfizer. Other topics include updates from ASH, the new Amgen CSO plus the group shares their thoughts on what to expect in 2024. *This episode aired on December 15, 2023

Howie and Harlan are joined by Tina Loarte Rodriguez, associate director, health equity measures, at Yale's Center for Outcomes Research & Evaluation and the author of Latinas in Nursing: Stories of Determination, Inspiration, and Trust. And Howie and Harlan discuss the clinical and economic dimensions of two newly approved CRISPR-based treatments for sickle cell disease.  Links: Tina Loarte Rodriguez Tina Loarte Rodriguez: Latinas in Nursing: Stories of Determination, Inspiration, and Trust Center for Outcomes Research & Evaluation (CORE) Center for Outcomes Research & Evaluation: Quality Measurement “Sociodemographic Disparities in Queue Jumping for Emergency Department Care” New Treatments for Sickle Cell Disease “In historic decision, FDA approves a CRISPR-based medicine for treatment of sickle cell disease” “F.D.A. Approves Sickle Cell Treatments, Including One That Uses CRISPR” The Nobel Prize in Chemistry 2020 Yale New Haven Health: Sickle Cell Program “Milestones in Sickle Cell Research and Care” Bluebird Bio Yahoo Finance: Bluebird Bio, Inc. “Tough road ahead for Bluebird Bio despite FDA approval for sickle cell therapy” “Harlan M. Krumholz Named Next Editor-in-Chief of JACC” Read an unedited transcript of this episode. Learn more about the MBA for Executives program at Yale SOM. Learn more about the Pozen-Commonwealth Fund Fellowship in Health Equity Leadership. Email Howie and Harlan comments or questions.

Howie and Harlan are joined by Tina Loarte Rodriguez, associate director, health equity measures, at Yale's Center for Outcomes Research & Evaluation and the author of Latinas in Nursing: Stories of Determination, Inspiration, and Trust. And Howie and Harlan discuss the clinical and economic dimensions of two newly approved CRISPR-based treatments for sickle cell disease.  Links: Tina Loarte Rodriguez Tina Loarte Rodriguez: Latinas in Nursing: Stories of Determination, Inspiration, and Trust Center for Outcomes Research & Evaluation (CORE) Center for Outcomes Research & Evaluation: Quality Measurement “Sociodemographic Disparities in Queue Jumping for Emergency Department Care” New Treatments for Sickle Cell Disease “In historic decision, FDA approves a CRISPR-based medicine for treatment of sickle cell disease” “F.D.A. Approves Sickle Cell Treatments, Including One That Uses CRISPR” The Nobel Prize in Chemistry 2020 Yale New Haven Health: Sickle Cell Program “Milestones in Sickle Cell Research and Care” Bluebird Bio Yahoo Finance: Bluebird Bio, Inc. “Tough road ahead for Bluebird Bio despite FDA approval for sickle cell therapy” “Harlan M. Krumholz Named Next Editor-in-Chief of JACC” Read an unedited transcript of this episode. Learn more about the MBA for Executives program at Yale SOM. Learn more about the Pozen-Commonwealth Fund Fellowship in Health Equity Leadership. Email Howie and Harlan comments or questions.

Drs. John Sweetenham and Fred Locke discuss the FDA investigation on the risk of cancer from CAR T-cell therapy and share insights on the known number of cases and the potential implications on clinical research and patient care. TRANSCRIPT Dr. John Sweetenham: Hello, I'm Dr. John Sweetenham from the UT Southwestern Harold C. Simmons Comprehensive Cancer Center and host of the ASCO Daily News Podcast. CAR T-cell therapies have been game changers for treating certain cancers including lymphomas and leukemias, as well as multiple myeloma, since the vast majority of patients who have received CAR-T do not have other curative options with conventional non-cellular, anti-cancer therapies. But on November 28, the U.S. Food and Drug Administration announced that it is investigating whether CAR T-cell therapy can, in rare cases, cause secondary cancers. The FDA launched the probe after receiving reports from clinical trials and other data sources of T-cell malignancies in patients who received CAR T-cell immunotherapies. Joining me to discuss the investigation and its implications for the field is Dr. Fred Locke, a medical oncologist and translational researcher and a senior member and chair in the Department of Blood and Marrow Transplant and Cellular Immunotherapy at the Moffitt Cancer Center in Tampa, Florida. Dr. Locke is an internationally renowned clinical research leader in the field of CAR T-cell therapy. You'll find our disclosures in the transcript of this episode, and disclosures of all guests on podcasts are available at asco.org/DNpod. Fred, it's great to have you on the podcast today. Dr. Fred Locke: Thanks, John, I'm really glad to be here. Dr. John Sweetenham: So, T-cells are the backbone of CAR T-cell therapies, and there are currently 6 CAR-T products approved by the FDA. As our listeners will know, CAR T-cell therapies manipulate a patient's T-cells, enabling them to recognize and attack antigens on cancer cells and induce potential long-standing changes in the immune system. In its statement on November 28, the FDA said it determined that the risk of T-cell malignancies is applicable to all of the currently approved BCMA- and CD19-directed, genetically modified, autologous CAR T-cell immunotherapies. Fred, could you comment for us on the investigation: How many patients are reported to have developed second malignancies from CAR T-cell therapy, and whether there are likely to be more secondary cancers reported? Dr. Fred Locke: Yes, so the FDA and the reports are coming out that there are 19 cases of T-cell malignancy that we're aware of that have occurred after the current FDA-approved CAR T-cell therapies were administered for the treatment of leukemia, lymphoma, or multiple myeloma. The majority of those cases were reported through the FDA Adverse Event Reporting System. And we don't know a lot of details of those 19 cases. We think that there's probably about 13,000 to 14,000 patients who've been treated with the commercial CAR T-cell therapies. So if you kind of do some crude math, you can come up with 19 out of say, 13,500; we're at about 0.1% of patients who could have developed T-cell lymphoma after treatment with these CAR T-cell therapies. It's not entirely out of the realm of possibility that T-cell lymphoma could develop from gene-modified T-cells, and these are all the patient's own T-cells that have been modified outside of the body. But I would still posit that this is a really low incidence of T-cell lymphomas in these patients who really are without other great treatment options. Dr. John Sweetenham: Yeah, and I think that's a point that we'll return to a little bit later on in the conversation around the fact that you know, clearly, there are major benefits that have been associated with CAR T-cell therapy and hematologic malignancies so far. And of course over the years, I think that many of us have become familiar with and learned a great deal about how to manage some of the more serious side effects of CAR T-cell therapy. And you, of course, have led several pivotal national trials of anti-CD19 CAR-Ts for lymphoma. Can you comment at all on whether you've seen previous data from your own practice or others on the risk of second malignancy from CAR T-cell therapy? And can you share your insights on the data and any new emerging data that warrants our attention for the concern or risk of second malignancy? And I guess to round up that series of questions, is there anything currently in yours or others research into CAR-T to explain what's happening and why this is going on? Dr. Fred Locke: I think what may have prompted the FDA's announcement of this is that on the same date that they came out with announcing their investigation, there was the release of the abstracts for the American Society of Hematology Annual Meeting. And within those abstracts, and unfortunately it was not selected for poster or oral abstract presentation, but discovered within those abstracts was one on a CAR+ T-cell lymphoma after ciltacabtagene autoleucel therapy for relapsed refractory multiple myeloma. And what these investigators and the company were reporting is that a patient with refractory multiple myeloma received the cilta-cel BCMA-directed CAR T-cell therapy and developed a stringent complete response, and about 5 months later developed a nasal facial plaque and PET+ cervical lymph nodes. And both the lymph nodes and the plaque were biopsied and showed a T-cell lymphoma in which 90% to 100% of the cells were positive by qPCR for the CAR construct and immunohistochemistry for the CAR protein. So this was a T-cell lymphoma growth where the cells were expressing the inserted protein, the chimeric antigen receptor protein, which is obviously not natural. And when they looked a little bit deeper at these patients, 91% of the cells have the same T-cell receptor sequence. So this was really a clonal sort of process. They did CAR integration analysis to see how the insertion of the CAR, the chimeric antigen receptor gene, could have potentially disrupted a gene within the T-cells. And what they found is that there were some dominant sort of insertions within certain genes suggesting monoclonality, but it wasn't within any sort of obvious activating genes that would be expected to lead to the T-cell lymphoma. They went on and did some additional analysis, and they showed that there was some existing TET2 mutations in the T-cells of this patient, prior to probably prior to the CAR T-cell manufacturer, and they weren't associated with clonal insertion. And I think, you know, it's possible that this patient who had a pre-existing mutation may have been susceptible to the development of a T-cell lymphoma prior to the CAR T-cell treatment. And TET2 was previously shown a number of years ago in a CLL patient treated with CD19 CAR T-cell therapy; it was shown that there was insertional mutagenesis, silencing the TET2 gene, and that associated with clonal expansion of the CAR T-cells in that patient and corresponded with remission of the CLL. However, the difference here is that that patient's T-cell clone went back down and contracted, and the patient remained in remission 5 years later with their T-cells still in the blood, but the minority of those T-cells had that that TET2 mutational insertional mutagenesis. All this is something we thought was theoretically possible, that T-cell lymphoma could develop after car T-cell therapy. And in fact, a prior trial using a different method of delivery of a CAR gene; instead of using a virus to insert the car into the into the T-cells, a transposon system called piggyBac was used. And in that trial, again, CD19 CAR trial, but in this case, it was allogeneic donor cells for patients who had relapsed after an allogeneic transplant. So it's sort of an autologous, you know, analogy, but it's using the donor cells. And in that trial, 2 out of like 10 patients developed clonal T-cell lymphoma, which was CAR+, but they weren't able to identify a clear insertional mutagenesis event in those cases. So, we've known this is possible, and it would have been great if this poster or if this abstract at ASH was presented as an oral or a poster so we could get more detail, but it's possible that that's the likely reason for the FDA's announcement. Dr. John Sweetenham: Thanks. The bottom line, I guess, is that for now, the jury's still out on exactly what's underlying these observations, but something which I'm sure is going to be the subject of a lot of discussion during the ASH meeting this year and moving forward. I'd like to inform our listeners that ASCO released a statement on the FDA investigation, stating that the risk of T-cell malignancies due to CAR T-cell therapy appears to be very low. And we've just heard from Dr. Locke that, of the several thousand patients who've received CAR Ts, there are 19 cases so far, it's been reported, which puts us into some type of proportion. The ASCO statement goes on to say that based on available data, and while such malignancies have occurred in patients who have received CAR T-cell therapy, the causal relationship, whether these cases are spontaneous or are caused by the therapy, needs to be investigated further, and we've just heard a little about the detail of that. ASCO added that by issuing a warning but not revoking approval of these therapies, the FDA clearly believes that the current available evidence suggests the overall benefits of these products, used within their approved label, continue to outweigh the potential risks. So Fred, the risk of secondary malignancies is already included as a class warning in the U.S. prescribing information for these CAR T-cell therapies. But do you think that the CAR-T products could eventually be taken off the market, and how would your research be impacted if this were to happen? And maybe finally, how long will patients on CAR T-cell therapy need to be monitored moving forward? Dr. Fred Locke: I don't believe that CAR T-cell therapy will be taken off the market. As we've already talked about, the incidence is extraordinarily low and the causality is unclear. It would certainly impact my research, as I'm doing clinical trials with CAR T-cell therapies, but it would more importantly impact the way we treat patients. We did over 300 CAR T-cell therapy treatments last year here at Moffitt Cancer Center. We're one of the busiest programs in the world giving CAR T-cell therapy, and it is truly a transformative therapy for all the diseases that we administer these FDA-approved therapies for. For example, in diffuse large B-cell lymphoma, we participated in the ZUMA-7 clinical trial and recently reported that patients randomized to CAR T-cell therapy had improved overall survival. They were living longer than patients randomized in the second-line setting to get conventional chemotherapy and autologous transplant. This is clearly a therapy that can work. I would also add that the risk of secondary malignancies is real, but that's a risk for all cancer patients, particularly patients with hematologic malignancies, and for example, lymphoma patients who've gotten an autologous stem cell transplant are at a relatively high lifetime risk of developing a secondary myeloid malignancy, most commonly, treatment-related MDS or AML. And that risk is also present after CAR T-cell therapy. The degree of attribution of CAR-T versus the condition of chemotherapy for CAR-T versus the previous chemotherapy is all unclear, and more analysis needs to be done. But the risk of developing treatment-related MDS or leukemia is certainly higher than the small number of T-cell lymphomas reported. The other thing I want to point out is that there was an analysis of the SEER database that patients with B-cell lymphomas are at about a 5-fold higher risk of developing a T-cell lymphoma than the otherwise healthy population; and vice versa, by the way, T-cell lymphoma patients are at risk for developing B cell lymphoma. And in fact, in that SEER database, it's not a wildly different percentage chance of developing a T-cell lymphoma after a B-cell lymphoma. And this data came out before the advent of CAR T-cell therapy. So I really think we need more science to be done to understand what's happening for these patients. Will this impact the field? Well, certainly, there are treatments that are not CAR T-cell therapy that compete with CAR T-cell therapy or could; I'm a strong believer that they don't offer the same outcomes for patients, but we will certainly see people talking about this for some time. Then the other place where this could be relevant, I think, is as we look at CAR T-cell therapy for autoimmune disorders, and we're starting to see studies of that for lupus and other diseases, the risk to benefit ratio could be different in those cases. So this is something we really need to consider as we move forward with CAR T- cell therapy. Dr. John Sweetenham: Yeah, thanks, Fred. And as a major clinical investigator in the field of CAR-T at the moment, do you see any potential concerns about difficulties in getting patients onto the trials of CAR T-cell in the light of this information? Dr. Fred Locke: No, I really don't. We're not seeing hesitancy, at least in the patients who are referred in for CAR T-cell therapy. Again, it may give ammunition for those who are already predisposed to not refer patients in for CAR T-cell therapy, but I don't think it should. I think that these are low risks, and these therapies clearly have benefits to patients. And we should give their patients an opportunity to get these therapies, and I don't see it impacting our clinical trials at this point. Dr. John Sweetenham: Yeah, and your comments address what was going to be my final question to you and that is, as a referring oncologist, how would you advise a referring oncologist to talk with their patients about these data and their implications moving forward? Dr. Fred Locke: If the patient brings it up, I think the response should be that these are very few cases of very low incidence and very low risk. There are other risks to CAR T-cell therapy that are greater, and really speaking with a cell therapist who administers the treatment is probably the best way to give the patient the option to get CAR T-cell therapy if they want to, knowing all the risks and benefits. So, I would leave it up to the CAR-T treatment center to discuss those risks with the patient.   Dr. John Sweetenham: Well, thanks, Fred, for sharing your insights with us on these concerning developments in CAR T-cell therapy, and I think also for putting them into context in terms of the sort of magnitude of this problem in the context of the overall number of patients who are benefiting from this therapy right now. We truly appreciate your time, and thanks for sharing your thoughts with our listeners. Dr. Fred Locke: Thanks, John, my pleasure. Dr. John Sweetenham: And thank you to our listeners for your time today. If you value the insights you hear on ASCO Daily News Podcast, please take a moment to rate, review, and subscribe wherever you get your podcasts. Disclaimer:  The purpose of this podcast is to educate and to inform. This is not a substitute for professional medical care and is not intended for use in the diagnosis or treatment of individual conditions. Guests on this podcast express their own opinions, experience, and conclusions. Guest statements on the podcast do not express the opinions of ASCO. The mention of any product, service, organization, activity or therapy should not be construed as an ASCO endorsement.    Find out more about today's speakers:      Dr. John Sweetenham Dr. Fred Locke @DrFredLocke     Follow ASCO on social media:         @ASCO on Twitter     ASCO on Facebook     ASCO on LinkedIn         Disclosures:        Dr. John Sweetenham:    Consulting or Advisory Role: EMA Wellness     Dr. Fred Locke: Consulting or Advisory Role: Novartis, Celgene, Calibr, Allogene, Gerson Lehrman Group, EcoR1, Amgen, Bluebird Bio, Bristol Myers Squibb, Iovance Biotherapeutics, Legend Biotech, Cowen, Kite (Gilean), Umoja Biopharma, Takeda, Sana Biotechnology, Daiichi Sankyo/UCB Japan, Bristol-Myers Squibb/Celgene, Janssen, A2 Biotherapeutics, Mittenyi Biotec, Caribou Biosciences, Takeda, Umoja Biopharma Research Funding: Kite Pharma, Allogene, Novartis, Bluebird Bio, Bristol-Myers Squibb/Calgene Patients, Royalties, Other Intellectual Property: Double Mutant Survivin Vaccine. US010414810B2 CAR T Cells with Enhanced Metabolic Fitness; Serial Number: 62/939,727 Methods of Enhancing CAR T Cell Therapies. Serial Number: 62/892,292. Evolutionary Dynamics of Non-Hodgkin Lymphoma CAR-T cell therapy.  Serial Number: 62/879,534. Travel, Accommodations, Expenses: Kite Pharma, A2 Biotherapeutics

On this week's episode of Biotech Hangout, hosts Daphne Zohar, Tim Opler, Josh Schimmer, Brian Skorney and guest Matthew Gline, Roivant CEO discuss the current market for public biotechs plus Pharma performance including Sanofi, Pfizer, Bristol Myers Squibb, Roche, Biogen and Abbvie. The hosts also discuss Sarepta's trial miss, and Matt shares additional commentary to Roivant's deal with Roche. They also share thoughts on Lexeo Therapeutics' IPO ($100M), 4D Molecular Therapeutics' CF Phase 1 data and AstraZeneca's investment in Cellectic ($245M). There were several large funds from the week including Abingworth's fundraise ($365M), Blue Owl- Cowen Investments acquisition (~$1B) and Bioluminescence Ventures' fundraise ($477M). Other topics include Bluebird Bio presells its PRV to Novartis, Amgen stops selling PSMA BiTE, Crispr Therapeutics/Vertex adcomm and Pfizer closes several sites. *This episode aired on November 3, 2023 Josh Schimmer disclosures: The disclosures for companies that I cover can be found on the Hangout homepage and none of the comments should be construed as investment advice.

Kristin Viswanathan Wolff, Vice President of Market Access at bluebird bio, joins us to discuss the cell and gene therapy landscape, why this is gene therapy's “moment,” and opportunities to advance collaboration across the healthcare industry to expand access and use of these innovative treatments.   To learn more about Healthscape, or to join the podcast, please contact Ellie Schwab or Pam Divack. 

With visions of a biology PhD dashed in a senior lab experiment gone way wrong, Andrew Obenshain envisioned a business career that would ultimately land him as CEO of a biotech firm. The industry being in its nascent days, there weren't many avenues there, but he found his way into a biotech consulting firm and later a biotech venture capital firm. Realizing if he wanted to be a CEO he'd need to know what CEO's do and realized they all had on-the-ground operations experience within a company. So to even get a foot in the door, he had to take some odd-looking sidesteps including taking a post business school job as a sales guy which he then parlayed to some other opportunities.His highly successful type A wife, who had her own career. And while paving the way for many of the family's moves, she made it possible for them to live out a dream to move with their kids to Paris. A period full of exploration and fun, however, was upended by a set of unexpected diagnoses.In this episode, find out from Andrew how finding joy can be possible even in the hardest times … on ROADS TAKEN...with Leslie Jennings Rowley. About This Episode's GuestAndrew Obenshain is CEO of Bluebird Bio, a biotech company focused on advancing therapies for rare diseases. He lives with his three kids in a suburb of Boston and is surrounded by the love of family and friends, present and past, at all times. (250) For another story about finding joy in parenthood and community despite loss, listen to our Christy Hansel Lohof. Find more episodes at https://roadstakenshow.comExecutive Producer/Host: Leslie Jennings RowleyMusic: Brian BurrowsEmail the show at RoadsTakenShow@gmail.com

In this episode, we talk with our panel about updates their company gave at the recent JP Morgan Healthcare Conference that took place earlier this year. This once-yearly conference is the epicenter for biotech deal-making and groundbreaking data releases. Dr. Robert Ross is the Chief Executive Officer of Surface Oncology. Surface Oncology's mission is to deliver transformative outcomes for people with cancer by breaking through the frontiers of immuno-oncology research. Before becoming CEO, Rob served as Chief Medical Officer at Surface. Prior to joining Surface, he led several programs in hematology and oncology at Bluebird Bio, including the anti-BCMA CAR T cell therapy program. Rob received his MD from Columbia University and has received extensive training in hematology and oncology at top institutions including the University of California San Francisco, Dana Farber, and Mass General Hospital.Dr. Paul Lammers is the Chief Executive Officer of Triumvira Immunologics. Triumvira is leveraging its proprietary T-cell antigen coupling technology to enhance engineered cell therapy to target solid tumors. Paul has a rich background in biotech management, having served as President & CEO at Mirna Therapeutics and Chief Medical Officer and Head of US Product Development for EMD Serono. Paul received his MD from Radboud University in the Netherlands.Hosted by Joe Varriale.

In this episode, Ayesha discussed the FDA approval of bluebird bio's Skysona for slowing the progression of neurologic dysfunction associated with the rare neurological disorder cerebral adrenoleukodystrophy (CALD) in boys four to 17 years of age with early, active CALD. The one-time administered gene therapy has also taken the title of being the world's most expensive drug at $3 million. Hear more about the treatment and how the high prices of gene therapies can be alarming, but reimbursement programs are designed to pick up the costs.Ayesha also talked about the emergence of a new COVID-19 variant called BF.7 that the CDC is closely watching. The new strain of the coronavirus is a subvariant of the BA.5 Omicron variant. It appears to be more transmissible than other variants and subvariants and its incidence has doubled in just two weeks according to the latest CDC data. Find out more about BF.7 including how it could drive the new anticipated wave of COVID infections this fall.Read the full articles here:Bluebird Bio's Skysona Receives FDA Approval and Becomes World's Most Expensive DrugCDC Expresses Concern Over New COVID-19 Variant BF.7For more life science and medical device content, visit the Xtalks Vitals homepage.Follow Us on Social MediaTwitter: @Xtalks Instagram: @Xtalks Facebook: https://www.facebook.com/Xtalks.Webinars/ LinkedIn: https://www.linkedin.com/company/xtalks-webconferences YouTube: https://www.youtube.com/c/XtalksWebinars/featured

First, STAT's Tara Bannow joins us to discuss how private equity's mounting interest in autism care has created an untenable situation for many parents in the U.S. We also explain the implications of Bluebird Bio's long-awaited FDA approval, a controversial treatment for ALS, and the ups and downs of Merck's reported interest in buying Seagen.

On this week's episode FirstWord Pharma PLUS editors Michael Flanagan and Simon King discuss why Gilead is getting flak for a positive cancer drug study, what's gone wrong at bluebird bio and why the FDA has been so quick to expand approval of Bristol Myers Squibb's Opdivo for the treatment of neoadjuvant lung cancer.

Today, we revisit one of our favorite episodes from 2021 featuring Michele Rhee, young adult cancer survivor of thyroid cancer, whose ordeal left her having over a dozen major surgeries, including open-heart surgery for a related underlying rare disease. Michele received both her MBA and MPH in three years and, because she's such an underachiever, went on to pursue a storied career representing the voice of the patient at every company she's worked for including The National Brain Tumor Society, Takeda Oncology, Bluebird Bio, and now as the VP of Patient Affairs at X4 Pharma. She's also traversed all seven continents in a quest to find herself, make sense of the madness, and live her life on her terms. For more information on us, visit https://OffScrip.com and follow @MZOutofPatients, @MatthewZachary, @VaxOnPod, and @OffScripMedia on Twitter. See Privacy Policy at https://art19.com/privacy and California Privacy Notice at https://art19.com/privacy#do-not-sell-my-info.

Sophie Schmitz and Joanna Fernandes discuss bluebird bio's innovative gene therapy Zynteglo and its withdrawal from the European market. Sophie and Joanna will be looking at the fall of Zynteglo from two sides: from a company perspective and from the perspective of the EU environment. Presenter: Georgie Rack, Communication Executive Contributors: Sophie Schmitz, Managing Partner and Joanna Fernandes, Senior Consultant Producer: Operations team

Pink Sheet reporters and editors discuss Bluebird Bio's decision to pull is gene therapy business from Europe, Eli Lilly potentially pushing regulatory boundaries with its Olympic TV spots, and the CMS decision to scrap the Trump Administration's most favored nation rule.

Scott Cleve brings an entire career dedicated to the mastery of global biopharma regulatory standards to his role as Vice President of Regulatory Operations and Compliance at bluebird bio. Join Cleve, host Matt Pillar, and Erin Harris, chief editor at Cell & Gene, for a candid discussion on the regulatory trends shaping the advance of bluebird's pipeline of gene therapies for the treatment of serious, life-altering diseases including Cerebral Adrenoleukodystrophy, Multiple Myeloma, Transfusion-Dependent β-Thalassemia, and Sickle Cell Disease.

FirstWord Pharma PLUS editors Michael Flanagan, Simon King and Becky Simon discuss the implications of two noteworthy news events in the field of gene therapy this week – bluebird bio's decision to walk away from Zynteglo reimbursement negotiations in Germany and Vertex's investment of $900m to increase its share of the economics in CTX001, a gene-editing sickle cell disease therapy being co-developed with CRISPR Therapeutics. They also discuss feedback from a key opinion leader who thinks Bristol Myers Squibb could be poised to lead the neoadjuvant non-small cell lung cancer market with its immunotherapy Opdivo and run the rule over Roche and Biogen's first-quarter results.

Studies have shown that chimeric antigen receptor (CAR) T-cell therapies produce responses in patients with relapsed/refractory B-cell lymphomas, but researchers continue to look for ways to improve efficacy, decrease toxicity, and overcome treatment resistance. Leslie Kean, MD, PhD, of Boston Children’s Hospital, discusses some of this research with host David H. Henry, MD, in this episode. Dr. Kean outlines four recent studies of CAR T-cell therapies in lymphoma. The studies were selected as part of the “Best of ASH” session at the 2020 annual meeting of the American Society of Hematology. Primary Analysis of ZUMA-5: A Phase 2 Study of Axicabtagene Ciloleucel (Axi-Cel) in Patients with Relapsed/Refractory Indolent Non-Hodgkin Lymphoma This study was designed to assess the efficacy and safety of axicabtagene ciloleucel (axi-cel) in patients with indolent lymphomas. In follicular lymphoma, the overall response rate (ORR) was 94%, and the complete response (CR) rate was 80%. In marginal zone lymphoma, the ORR was 85%, and the CR rate was 60%. There was one grade 5 and one grade 4 case of cytokine release syndrome (CRS). Dr. Kean noted that 146 patients were evaluable for adverse events, so the single death related to CRS should be viewed in that context. Overall, 82% of patients had CRS of any grade. Jacobson C et al. ASH 2020, Abstract 700. https://bit.ly/32at91V. What’s involved in a CAR T-cell study? Dr. Kean explained that a patient is first deemed eligible by an oncologist and then enrolled in a CAR T-cell study. For studies like ZUMA-5 that are testing autologous CAR T cells, basic lab work is done to ensure the patient has a high enough lymphocyte count. The patient then undergoes apheresis, and the patient’s T cells are used to create the CAR T-cell product. The company developing the product transduces the T cells with the CAR so the resulting CAR T cells will target cancer cells. The therapy in ZUMA-5, axi-cel, targets CD19, which is expressed on B-cell lymphoma cells. Normal B cells express CD19 as well, so immunoglobulin replacement is sometimes necessary to offset the loss of normal B cells. Efficacy and Safety of Tisagenlecleucel in Adult Patients with Relapsed/Refractory Follicular Lymphoma: Interim Analysis of the Phase 2 ELARA Trial Tisagenlecleucel differs from axi-cel in the signaling domain, though tisagenlecleucel targets CD19 as well, Dr. Kean explained. She noted that tisagenlecleucel is a bit more long-lived than axi-cel. In this trial, tisagenlecleucel produced an ORR of 82% and a CR rate of 65%. There were no cases of grade 3 or higher CRS, which may be attributed to the different signaling domain, Dr. Kean said. Fowler NH et al. ASH 2020, Abstract 1149. https://bit.ly/2OIGjjA. TRANSCEND CLL 004: Phase 1 Cohort of Lisocabtagene Maraleucel (liso-cel) in Combination with Ibrutinib for Patients with Relapsed/Refractory (R/R) Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma (CLL/SLL) Patients in this study received the CAR T-cell therapy liso-cel in combination with the BTK inhibitor ibrutinib. The combination increased both efficacy and safety, as ibrutinib assisted in calming down the immune response. There were no grade 5 adverse events and no cases of grade 4 CRS or neurotoxicity. The ORR was 95%, and the CR rate was 63%. There was no difference in response among patients who had or had not received a BTK inhibitor previously, Dr. Kean noted. Wierda WG et al. ASH 2020, Abstract 544. https://bit.ly/3uPuJ5U. CD58 Aberrations Limit Durable Responses to CD19 CAR in Large B Cell Lymphoma Patients Treated with Axicabtagene Ciloleucel But Can Be Overcome Through Novel CAR Engineering Dr. Kean noted that CAR T-cell therapy typically produces a CR in more than 90% of patients within 30 days, but the long-term duration of response is about 50%. With this study, researchers wanted to investigate why a CAR T-cell therapy would fail and determine if any tumor-specific factors affect the duration of response. The team found that patients who had mutations in CD58 were less likely to achieve a CR to axi-cel, and most patients with these mutations ultimately progressed. CD2, the T-cell ligand for CD58, plays adhesive and costimulatory roles in T cells, and CAR T cells rely on intrinsic T-cell signaling to work, Dr. Kean explained. So if the CD2 in a CAR T cell can’t “see” CD58 on the tumor because of a mutation, the CAR T cell doesn’t work, she added. To bypass this, the researchers created a construct integrating CD2 costimulatory domains within the CAR molecule so it expressed CD2 in a way that doesn’t require CD58. The new construct “cures tumors like gangbusters” in mouse models, Dr. Kean said, adding that this CAR T-cell therapy could be coming to the clinic soon. Maizner RG et al. ASH 2020, Abstract 556. https://bit.ly/3283zL0. Looking ahead: Concerns about cost Cost is a critical barrier to receiving CAR T-cell therapy, Dr. Kean noted, especially for patients who require additional treatment after receiving CAR T cells. The next generation of CAR T-cell research should determine if this treatment is best used as a bridge to transplant or if CAR T-cell therapy can stand alone, she added. To make the cost more palatable, CAR T-cell products should be a final cure, Dr. Kean said. Show notes written by Malika Gill, MD, a resident at Pennsylvania Hospital, Philadelphia. Disclosures Dr. Henry has no relevant disclosures. Dr. Kean disclosed relationships with Magenta Therapeutics, Bristol-Myers Squibb, Kymab, HiFiBiO Therapeutics, Regeneron, Novartis, Gilead, Bluebird Bio, and Forty Seven. *  *  * For more MDedge Podcasts, go to mdedge.com/podcasts Email the show: podcasts@mdedge.com Interact with us on Twitter: @MDedgehemonc David Henry on Twitter: @davidhenrymd

Sangamo Therapeutics is clinical-stage biotech company looking to commercialize a number of assets for the treatment of disease. Their furthest along program is a gene therapy treatment for Hemophilia A. The sector has seen some recent upsets with the FDA issuing a CRL to Biomarin for their HemA gene therapy, as well as some safety concerns with Uniqure and Bluebird Bio. Sangamo also has a number Phase 1/2 programs that will have readouts in late 2021 as well as a large preclinical pipeline. They have been able to leverage a number of partnerships with Pharmaceutical companies that has led to significant cash payments and equity purchases. In this episode, I share an email exchange I had with the SVP, Head of Business Development at Sangamo, Melita Sun Jung. I also discuss updates from BioXcel and Bluebird Bio. Check out our sponsor at Gallant.com and use promo code: BIO to save when you enroll your pet for stem cell banking. Presentation slides: https://drive.google.com/drive/folders/1MENe3kFzFosR6-ALPoY9ovpXXcERH_j3?usp=sharing Help out the show (or join the discord) by becoming a patron at: https://www.patreon.com/breakingbiotech Follow me on twitter @matthewlepoire Send me an email matthewlepoire@gmail.com www.breakingbiotech.com #breakingbiotech Disclaimer: All opinions expressed by Matt in this podcast are solely his opinions. You should not treat any opinion expressed by Matt in this podcast as a specific inducement to make a particular investment or follow a particular strategy, but only as an expression of his opinion. Matt's opinions are based upon information he considers reliable, but Matt cannot warrant its completeness or accuracy, and it should not be relied upon as such. Matt is not under any obligation to update or correct any information provided in this podcast. Past performance is not indicative of future results. Matt does not guarantee any specific outcome or profit. You should be aware of the real risk of loss in following any strategy or investment discussed in this podcast. #biotech

FirstWord Pharma PLUS editor Simon King discusses the approval of Bristol Myers Squibb and bluebird bio's CAR-T therapy Abecma for multiple myeloma with senior Therapy Trends analyst Sarah Harris, speaks to Novartis' European gene therapy chief Mike Fraser about the launch of Zolgensma and asks FirstWord HealthTech's Tina Tan why the US Federal Trade Commission is looking to block Illumina's proposed acquisition of Grail in the cancer diagnostics market.

Walmart shares slip on earnings. The Trade Desk surges on record revenue. Roku rises on an unexpected profit. Fastly falls on growth concerns. Shopify slips. CVS Health treads water. Berkshire-Hathaway makes some big investments. And Marriott suffers a big loss with the death of its CEO, Arne Sorenson. Motley Fool analysts Ron Gross and Jason Moser discuss those stories and weigh in on autonomous driving, big tech break-ups, and the streaming wars. Plus, Ron and Jason share two stocks on their radar: Bluebird Bio and RadNet.

We discuss all that and more this week on “The Readout LOUD,” STAT's biotech podcast. First, we break down a significant setback for Bluebird Bio's gene therapy program with some help from Akshay Sharma, a bone marrow transplant expert at St. Jude Children's Research Hospital. Then, STAT's Kate Sheridan joins us to discuss her deep dive into Flagship Pioneering, the superlatively successful and frequently grandiose venture firm behind Moderna. Finally, we dig into what the future might hold for a post-Trump FDA, which remains without a permanent commissioner.

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Dilan Chudasma is a Manufacturing Associate II at a small-medium sized gene therapy company. He graduated from the Joint Program of Biomedical Engineering at UNC and NC State in 2019. Dilan talks about manufacturing in pharma, gene therapy, and what he does every day. For more information on The BME Grad Podcast, visit the Joint Department of BME at UNC and NC State's website: bme.unc.edu/home/news-events/the-bme-grad-podcast/ Connect with or reach out to Host, Allie Mitzak, on LinkedIn: www.linkedin.com/in/allie-mitzak

Dilan Chudasma is a Manufacturing Associate II at a small-medium sized gene therapy company. He graduated from the Joint Program of Biomedical Engineering at UNC and NC State in 2019. Dilan talks about manufacturing in pharma, gene therapy, and what he does every day. For more information on The BME Grad Podcast, visit the Joint Department of BME at UNC and NC State's website: bme.unc.edu/home/news-events/the-bme-grad-podcast/ Connect with or reach out to Host, Allie Mitzak, on LinkedIn: www.linkedin.com/in/allie-mitzak

Part 2 of Anthony and Michael's meet up to Nina Bauer and Lance Holland. *Opinions expressed are solely the interviewee's own and do not express the views or opinions of their respective employers.*   The bulk of the chat was around Mesoblast's pending FDA decision for Ryoncil, their pediatric graft versus host disease therapy. The panel took it to a vote on will it be approved or rejected. Listen and find out how the panel judged it and of course, you can see the widely reported outcome in various news outlets.   Register for FREE for Phacilitate's next member event! Advanced Therapies Connect will take place on the 26-27 January 2021 and will be 100% virtual! https://www.advancedtherapiesconnect.com/   Lance's link to Mass Bio Ed https://www.massbioed.org   Dark Horse Consulting are recruiting! https://darkhorseconsultinggroup.com/careers/   And so is Nina's team at MilliporeSigma! https://www.merckgroup.com/en/careers.html   Finally, if you're not already, don't forget to sign-up as a Phacilitate member, it's FREE! Every fortnight you'll get the latest market intelligence, new ideas and exclusive event offers straight to your inbox.   Join a community with the same goal; to help advance a new standard in healthcare. https://www.phacilitate.co.uk/membership  

On the show today, I welcome Michelle Rhee, young adult cancer survivor of Thyroid Cancer (you know, “the good one” I’M KIDDING) whose ordeal left her having over a dozen major surgeries, including open heart surgery for a related underlying rare disease. Back in the heyday when the young adult cancer movement was taking off, Michele was looking to take an active role in our startup culture. After being introduced, I helped land her an internship at the Children’s Cause for Cancer Advocacy, a landmark organization that everyone should know about, which helped launch my career in founding Stupid Cancer. She received BOTH her MBA and MPH in three years and, because she’s such an underachiever, went on to pursue a storied career thus far representing the voice of the patient at every company she’s worked for; including The National Brain Tumor Society, Takeda Oncology, Bluebird Bio, and now as the VP of Patient Affairs at X4 Pharma. She’s also traversed ALL SEVEN continents in a quest to find herself, make sense of the madness, and live her life on her terms. So, prepare yourself for an inspirational conversation amongst friends, and if my “conversation amongst friends” I mean “eavesdropping on our first phone call in 7 years,” well, then you’re in for a treat. Learn more about the Children’s Cause for Cancer Advocacy

In this episode, Anthony and Michael talk to Nina Bauer, the Head of Commercial, Gene Editing and Novel Modalities at MilliporeSigma and Lance Holland, Associate Director, External Vector Manufacturing at bluebird bio.   The discussion starts with a look at the challenges brought by Covid-19 with regards to finding a new way of working with your CDMO, in particular conducting audits. Anthony's solution? Use GoPros!   Nina puts a question to the group around recruiting diverse talent. With a major talent gap across our industry, this surely has to have many benefits?   The conversation flowed so well that we ran out of time, keep your eye out for part II of this conversation coming soon...   If you're not already, don't forget to sign-up as a Phacilitate member, it's FREE! Every fortnight you'll get the latest market intelligence, new ideas and exclusive event offers straight to your inbox. Join a community with the same goal; to help advance a new standard in healthcare. https://www.phacilitate.co.uk/membership

The small, Chicago-based biotech venture Errant Gene Therapeutics, which was engaged in a court battle against a well-funded biotech called Bluebird Bio and a venture capital firm known as Third Rock Ventures. With allegations of breach of contract, tortious business interference, and fraud, TrialSite News initiated a project to review publicly available information, including court records, to derive a more thorough understanding of the case at hand. First podcast is located here

The small, Chicago-based biotech venture Errant Gene Therapeutics, LLC was engaged in a court battle against a well-funded biotech called Bluebird Bio (which has raised over $400 million) and a venture capital firm known as Third Rock Ventures. With allegations of breach of contract, tortious business interference, and fraud, TrialSite News initiated a project to review publicly available information, including court records, to derive a more thorough understanding of the case at hand.  In this podcast we ask Mr. Girondi about how this all started.

Business Radio Special: Host Joey Zwillinger, Wharton Alum and Co-Founder/Co-CEO of Allbirds, talks with Nick Leschly, CEO of Bluebird Bio, about their mission to heal people on a genetic level with targeted gene therapy on Purpose Built.Tune in on Business Radio SiriusXM 132 and Insights SiriusXM 121 for more great content. See acast.com/privacy for privacy and opt-out information.

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Bloomberg Markets with Carol Massar and Cory Johnson. GUESTS: Dani Burger Stocks Reporter Bloomberg News Discussing her story "For Quants, Megacap Tech Rout Looks Like a Systematic Unwind." David Wilson Stocks Editor Bloomberg News Discussing his "Stock of the Day"Bluebird Bio (BLUE). Bluebird Bio Inc.'s shares fell for the first time in eight days after a cancer therapy it's pursuing suffered a setback. Novartis AG treatment a study of the treatment for a form of leukemia because it wasn't effective, according to a U.S. government website that tracks drug research. Bluebird Bio is pursuing the therapy to treat a blood cancer, called multiple myeloma. The stock had surged 48 1/2 percent in the previous seven trading days.

Bloomberg Markets with Carol Massar and Cory Johnson. GUESTS: Dani Burger Stocks Reporter Bloomberg News Discussing her story "For Quants, Megacap Tech Rout Looks Like a Systematic Unwind." David Wilson Stocks Editor Bloomberg News Discussing his "Stock of the Day"Bluebird Bio (BLUE). Bluebird Bio Inc.'s shares fell for the first time in eight days after a cancer therapy it's pursuing suffered a setback. Novartis AG treatment a study of the treatment for a form of leukemia because it wasn't effective, according to a U.S. government website that tracks drug research. Bluebird Bio is pursuing the therapy to treat a blood cancer, called multiple myeloma. The stock had surged 48 1/2 percent in the previous seven trading days. Learn more about your ad-choices at https://www.iheartpodcastnetwork.com

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