Shows
TechNation Radio PodcastEpisode 25-29 Unlearning SilenceOn this week’s Tech Nation, Moira speaks with Author Elaine Lin Hering, who joins me to talk about her book, “Unlearning Silence … How to Speak Your Mind, Unleash Talent, and Live More Fully.” Then, Dr. Neil Kumar, Founder and CEO of BridgeBio Pharma, explains why we can expect genetic diseases to be solvable in our lifetime.2025-07-2559 min
TechNation Radio PodcastEpisode 25-29 Unlearning SilenceOn this week’s Tech Nation, Moira speaks with Author Elaine Lin Hering, who joins me to talk about her book, “Unlearning Silence … How to Speak Your Mind, Unleash Talent, and Live More Fully.” Then, Dr. Neil Kumar, Founder and CEO of BridgeBio Pharma, explains why we can expect genetic diseases to be solvable in our lifetime.2025-07-2559 min
BioTech Nation ... with Dr. Moira GunnSolving Genetic Diseases??? Dr. Neil Kumar, Founder & CEO, BridgeBio PharmaThis week on Biotech Nation, Dr. Neil Kumar, founder and CEO of BridgeBio Pharma. Dr. Kumar emphasizes that genetic diseases, particularly Mendelian diseases caused by single gene mutations, are solvable due to advancements in genetic measurement and understanding. He discusses BridgeBio Pharma's innovative corporate structure, which consists of multiple specialized companies under a centralized infrastructure to efficiently tackle various genetic diseases. This model allows for focused expertise on individual conditions while sharing common resources. Dr. Kumar highlights the company's commitment to using diverse therapeutic modalities, such as small molecules and gene therapies, to develop treatments that address the underlying...2025-07-2323 min
On Rare“It’s important to stay connected” Daniel is living with Tuberous Sclerosis Complex (TSC)Seizures, sleepless nights, and mysterious white patches on his skin marked the beginning of Daniel’s journey with Tuberous Sclerosis Complex (TSC). Diagnosed at age 6, Daniel faced a childhood filled with MRIs, EEGs, and specialist visits, often requiring cross-country travel for coordinated care. In this moving episode of On Rare, David Rintell, Head of Patient Advocacy at BridgeBio, and Mandy Rohrig, Senior Director of Patient Advocacy at BridgeBio Gene Therapy, speak with Daniel, a 31-year-old living in Seattle, about growing up with TSC, the emotional toll of visible symptoms, and the stigma he faced from peers. He reflects on th...2025-06-2729 min
Biotech HangoutEpisode 140 - May 2, 2025On this week’s episode, Eric Schmidt and Sam Fazeli are joined by special guests Adam Feuerstein and Peter Kolchinsky to discuss the upward movement of the biotech market along with strong drug launches, deals, and net-positive Q1 earnings, all signaling things may be improving. Next, the group notes the FDA is unlikely to undergo significant reorganization and while this stability is reassuring, complacency should be avoided as headwinds persist. Despite the turmoil, the pharmaceutical industry remains resilient, as people prioritize their health and the need for medicine. The conversation shifts back to the new FDA leadership, highlighting Makary an...2025-05-0858 minOn Rare“We just thought we were clumsy”. Katie and Allie are living with late-onset Tay-Sachs diseaseKatie and Allie’s story began in childhood with unexplained clumsiness and subtle symptoms that intensified over time. After years of searching for answers, Katie was diagnosed with late-onset Tay-Sachs (LOTS), a rare neurodegenerative disease. Further testing confirmed her twin sister Allie’s diagnosis as well. Despite facing daily challenges ranging from mobility issues to emotional strain, the sisters have become passionate advocates, raising over $1 million for research and awareness. With humor, grit, and the support of their family—especially their powerhouse mom—they continue to live fully and inspire the rare disease community.
In this moving episode...2025-04-2540 min
BioSpaceTrump’s Pharma Tariffs, Monarez for CDC, Novo’s New Obesity Play, MorePresident Donald Trump doubled down on tariff threats targeting pharma, saying additional levies on pharmaceuticals will come “at some point,” per CNBC. Meanwhile, Johnson & Johnson became the latest big pharma to respond to Trump’s warning of potential tariffs if companies don’t reshore their manufacturing, announcing a massive $55 billion U.S. manufacturing and R&D investment. Not all companies are on board, however: AstraZeneca is looking eastward, pumping $2.5 billion into a new research facility in Beijing. Also on the policy front, Trump nominated acting CDC director Susan Monarez for the top job after pulling his first no...2025-03-2619 minOn Rare"We say it all the time: We're lucky." Philip is living with hereditary transthyretin amyloidosis (ATTR)Philip’s journey with ATTR began with unexplained weight loss and gastrointestinal issues that puzzled doctors for years. Despite seeing multiple specialists, his condition remained undiagnosed until a physician at Johns Hopkins finally connected the dots, confirming ATTR. By then, Philip had lost 60 pounds and was on the brink of survival.
In today’s episode of On Rare, David Rintell, Head of Patient Advocacy at BridgeBio, and Mandy Rohrig, Senior Director of Patient Advocacy at BridgeBio Gene Therapy, speak with Philip and his wife, Sally, who has helped to navigate the frustrating medical system, advocate for answers, and...2025-03-2539 min
BioSpaceSarepta’s Elevidys Death, FDA’s Vaccine Move, Weldon Whiplash, PDUFAs in CardioA patient with Duchenne muscular dystrophy taking Sarepta’s gene therapy Elevidys has died of acute liver failure, possibly related to a recent viral infection. Sarepta, which said it will update Elevidys’ label to reflect the new safety signal, saw its shares drop 22% on the news but analysts still seem positive on the drug, as treatment options for Duchenne remain limited.Meanwhile, both AstraZeneca and Taiho Pharmaceuticals announced acquisitions worth up to $1 billion or more in two sizzling therapeutic spaces, cell therapy and antibody-drug conjugates, respectively.Despite canceling a vaccine advisory committee late last month, the...2025-03-1919 minOn Rare“There’s always somebody to fight for.” Kady’s son Julien is living with Autosomal Dominant Hypocalcemia Type 1 (ADH1)When Julien was just 6 weeks old, Kady knew something wasn’t right. Despite more than 16 doctors insisting Julien was fine, Kady trusted her instincts and fought for answers—refusing to leave the hospital until the right tests were done. That persistence led to a life-changing diagnosis: a rare genetic form of hypoparathyroidism. Julien’s specific mutation is so unique that he is the only known case in the world. In today’s episode of On Rare, David Rintell, Head of Patient Advocacy at BridgeBio, and Mandy Rohrig, Director of Patient Advocacy at BridgeBio Gene Therapy, speak with Kady, the mother o...2025-02-1842 min
PeerView Internal Medicine CME/CNE/CPE Video PodcastMichelle Kittleson, MD, PhD / Senthil Selvaraj, MD, MS, MA - Don’t Fail Them Now: Bridging Diagnostic and Treatment Gaps in Transthyretin Cardiac AmyloidosisThis content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/NCPD/CPE information, and to apply for credit, please visit us at PeerView.com/QTM865. CME/NCPD/CPE credit will be available until January 22, 2026.Don’t Fail Them Now: Bridging Diagnostic and Treatment Gaps in Transthyretin Cardiac Amyloidosis[JAC_Logo] In support of improving patient care, PVI, PeerView Institute for Medical Education, is jo...2025-01-2259 min
PeerView Clinical Pharmacology CME/CNE/CPE Audio PodcastMichelle Kittleson, MD, PhD / Senthil Selvaraj, MD, MS, MA - Don’t Fail Them Now: Bridging Diagnostic and Treatment Gaps in Transthyretin Cardiac AmyloidosisThis content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/NCPD/CPE information, and to apply for credit, please visit us at PeerView.com/QTM865. CME/NCPD/CPE credit will be available until January 22, 2026.Don’t Fail Them Now: Bridging Diagnostic and Treatment Gaps in Transthyretin Cardiac Amyloidosis[JAC_Logo] In support of improving patient care, PVI, PeerView Institute for Medical Education, is jo...2025-01-2259 min
PeerView Heart, Lung & Blood CME/CNE/CPE Audio PodcastMichelle Kittleson, MD, PhD / Senthil Selvaraj, MD, MS, MA - Don’t Fail Them Now: Bridging Diagnostic and Treatment Gaps in Transthyretin Cardiac AmyloidosisThis content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/NCPD/CPE information, and to apply for credit, please visit us at PeerView.com/QTM865. CME/NCPD/CPE credit will be available until January 22, 2026.Don’t Fail Them Now: Bridging Diagnostic and Treatment Gaps in Transthyretin Cardiac Amyloidosis[JAC_Logo] In support of improving patient care, PVI, PeerView Institute for Medical Education, is jo...2025-01-2259 min
PeerView Heart, Lung & Blood CME/CNE/CPE Video PodcastMichelle Kittleson, MD, PhD / Senthil Selvaraj, MD, MS, MA - Don’t Fail Them Now: Bridging Diagnostic and Treatment Gaps in Transthyretin Cardiac AmyloidosisThis content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/NCPD/CPE information, and to apply for credit, please visit us at PeerView.com/QTM865. CME/NCPD/CPE credit will be available until January 22, 2026.Don’t Fail Them Now: Bridging Diagnostic and Treatment Gaps in Transthyretin Cardiac Amyloidosis[JAC_Logo] In support of improving patient care, PVI, PeerView Institute for Medical Education, is jo...2025-01-2259 min
PeerView Internal Medicine CME/CNE/CPE Audio PodcastMichelle Kittleson, MD, PhD / Senthil Selvaraj, MD, MS, MA - Don’t Fail Them Now: Bridging Diagnostic and Treatment Gaps in Transthyretin Cardiac AmyloidosisThis content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/NCPD/CPE information, and to apply for credit, please visit us at PeerView.com/QTM865. CME/NCPD/CPE credit will be available until January 22, 2026.Don’t Fail Them Now: Bridging Diagnostic and Treatment Gaps in Transthyretin Cardiac Amyloidosis[JAC_Logo] In support of improving patient care, PVI, PeerView Institute for Medical Education, is jo...2025-01-2259 min
PeerView Clinical Pharmacology CME/CNE/CPE VideoMichelle Kittleson, MD, PhD / Senthil Selvaraj, MD, MS, MA - Don’t Fail Them Now: Bridging Diagnostic and Treatment Gaps in Transthyretin Cardiac AmyloidosisThis content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/NCPD/CPE information, and to apply for credit, please visit us at PeerView.com/QTM865. CME/NCPD/CPE credit will be available until January 22, 2026.Don’t Fail Them Now: Bridging Diagnostic and Treatment Gaps in Transthyretin Cardiac Amyloidosis[JAC_Logo] In support of improving patient care, PVI, PeerView Institute for Medical Education, is jo...2025-01-2259 minOn RareAnother year of incredible conversations! On Rare celebrates our 2024 podcast guests!In our final episode of 2024, On Rare looks back at highlights from the rewarding and rare conversations with our exceptional guests and David Rintell, Global Head of Patient Advocacy at BridgeBio and Mandy Rohrig, Director of Patient Advocacy at BridgeBio Gene Therapy. We listened, we learned, we laughed, and we cried. We are deeply grateful to all of our guests. Join us for a look back at 2024 and don’t forget to subscribe to learn more from our guests in 2025.
2025-01-0337 minOn Rare“When numbness of the hands is a window to the heart,” Charles is living with Transthyretin amyloidosis cardiomyopathy (ATTR-CM).Charles, a recently retired cardiologist of 53 years, speaks with David Rintell, Head of Patient Advocacy at BridgeBio, and Mandy Rohrig, Director of Patient Advocacy at BridgeBio Gene Therapy, about how his unexpected early diagnosis of ATTR-CM likely impacted the trajectory of his disease. Dr. Jonathan Fox, President and Chief Medical Officer of cardiorenal programs at BridgeBio, explains how ATTR affects the heart and peripheral nervous system, and the importance of early diagnosis.
For years, Charles suffered from numbness and tingling in both wrists and hands. Eventually, when the pain became so severe that operating a mouse for 10 to...2024-12-0933 minOn Rare“You can do everything that you want to do.” Erin is living with hypochondroplasia.Erin joins David Rintell, Head of Patient Advocacy at BridgeBio, and Mandy Rohrig, Director of Patient Advocacy at BridgeBio Gene Therapy, to share her experience living with hypochondroplasia. Erin also has two sons living with hypochondroplasia and one son of average height. Raised in a loving and supportive family with a can-do attitude, her father told her that she may be of short stature but that she could do everything she wanted to do, just maybe in a slightly different way.
Erin shares her diagnostic journey with hypochondroplasia in the third grade. Even though her diagnosis was...2024-10-2939 minOn Rare“I have 2I, but I am not 2I.” Misty’s Journey Living With LGMD2I/R9In today’s episode of On Rare, David Rintell, Head of Patient Advocacy at BridgeBio, and Mandy Rohrig, Director of Patient Advocacy at BridgeBio Gene Therapy, speak with Misty about her experience living with limb-girdle muscular dystrophy, type2I/R9 (LGMD2I/R9). Misty details the suffering she experienced in her childhood from bullying for the symptoms she now realizes are connected to LGMD2I/R9 and shares the many frustrations and significant challenges that resulted from her delayed diagnosis. Despite noticing alarming symptoms as early as junior high school, Misty was dismissed by medical professionals for years. Finally, an...2024-10-0345 minOn Rare“They told me to go home and love my child” Dawn’s daughter Vayle is living with Canavan Disease.In this emotional episode of On Rare, David Rintell, Head of Patient Advocacy at BridgeBio, and Mandy Rohrig, Director of Patient Advocacy at BridgeBio Gene Therapy, speak with Dawn, the single parent of Vayle, a 5-year-old girl living with Canavan Disease. Dawn shares how she came to realize that Vayle was not meeting developmental milestones at 3-4 months old. Despite initial reassurance from her pediatrician, Dawn’s maternal intuition proved correct, leading to a diagnosis of Canavan Disease during Vale’s 6-month checkup. Dawn was told that Vayle might not live beyond the age of ten.
Dawn reco...2024-08-1234 minOn Rare“I chose to reinvent myself.” Sean is living with hereditary transthyretin amyloidosis (ATTR).Sean joins David Rintell, Head of Patient Advocacy at BridgeBio, and Mandy Rohrig, Director of Patient Advocacy at BridgeBio Gene Therapy to share his journey with ATTR. In this episode, Sean describes his initial symptoms, which included carpal tunnel syndrome and numbness in his foot and ankle, the misdiagnosis he experienced, and his eventual diagnosis at the Cleveland Clinic in Abu Dhabi. While the diagnosis was a relief it was also difficult to accept and took an emotional toll on him and those around him. Sean has found this to be a common experience of many diagnosed with ATTR...2024-07-0930 minTechNation Radio PodcastEpisode 24-27 Unlearning SilenceOn this week’s Tech Nation, Moira speaks withAuthor, Elaine Lin Hering, joins me to talk about her book, “Unlearning Silence … How to Speak Your Mind, Unleash Talent, and Live More Fully.” Then, Dr. Neil Kumar, Founder and CEO of BridgeBio Pharma, explains why we can expect genetic diseases to be solvable in our lifetime.2024-07-0559 minTechNation Radio PodcastEpisode 24-27 Unlearning SilenceOn this week’s Tech Nation, Moira speaks withAuthor, Elaine Lin Hering, joins me to talk about her book, “Unlearning Silence … How to Speak Your Mind, Unleash Talent, and Live More Fully.” Then, Dr. Neil Kumar, Founder and CEO of BridgeBio Pharma, explains why we can expect genetic diseases to be solvable in our lifetime.2024-07-0559 minOn Rare“If I had known then what I know now, I wouldn’t have been so scared.” Alex is the mother of Hudson, a boy living with hypochondroplasia, a rare form of dwarfism.Alex joins David Rintell, Head of Patient Advocacy at BridgeBio, and Mandy Rohrig, Director of Patient Advocacy at BridgeBio Gene Therapy, to speak about her son, Hudson. Before Hudson’s birth, Alex and her husband learned that Hudson would be born with hypochondroplasia. A genetic counselor helped her to understand the diagnosis. Although Alex is a medical professional, she had never heard of this condition, and the many unknowns of this new diagnosis left Alex feeling scared. However, all these fears were put aside when she held Hudson for the first time. At five days old, Hudson had breathing pr...2024-06-1043 minOn Rare“If I had known then what I know now, I wouldn’t have been so scared.” Alex is the mother of Hudson, a boy living with hypochondroplasia, a rare form of dwarfism.Alex joins David Rintell, Head of Patient Advocacy at BridgeBio, and Mandy Rohrig, Director of Patient Advocacy at BridgeBio Gene Therapy, to speak about her son, Hudson. Before Hudson’s birth, Alex and her husband learned that Hudson would be born with hypochondroplasia. A genetic counselor helped her to understand the diagnosis. Although Alex is a medical professional, she had never heard of this condition, and the many unknowns of this new diagnosis left Alex feeling scared. However, all these fears were put aside when she held Hudson for the first time. At five days old, Hudson had breathing pr...2024-06-0743 min
Harvard Alumni Entrepreneurs InvitesDeep Tech: Money MattersIN THIS EPISODE: In this podcast episode, host Philip Guarino speaks with Andrew Lo, a distinguished professor at MIT and an entrepreneur with a focus on healthcare and deep tech ventures. Andrew shares his journey from academia to entrepreneurship, detailing his transition from economics to founding a quantitative investment firm and later exploring the financial challenges of drug development. He discusses the fundraising challenges for deep tech entrepreneurs, how investors’ risk calculation differs for more complex ventures and how this requires continuous investor communication and learning. Lo also highlights the vibrant innovation ecosystem in the Boston-Cambridge area an...2024-05-2126 minOn RareJonathan Fox, MD, talks about ATTR2024-04-2520 minOn Rare“We Only Live in This Moment.” Part Two of a two-part conversation with Eric, who lives with Transthyretin Amyloidosis (ATTR).In part two, Eric continues talking about his experience living with Transthyretin Amyloidosis (ATTR) with David Rintell, Head of Patient Advocacy at BridgeBio, and Mandy Rohrig, Director of Patient Advocacy at BridgeBio Gene Therapy. Eric recalls the surprise he experienced when a routine doctor’s visit led to him, within hours, to undergoing a heart transplant. Though the surgery was successful, Eric describes his complex recovery from the transplant. Age, old sports injuries, and lingering ATTR symptoms all contributed to a year-long recovery process, which included a two-week stay in the hospital, followed by an acute rehabilitation facility, a fe...2024-04-2521 min
Conversations at BasecampTaking Strategic Risks to Advance Your CareerReady to Make Bold Moves? This week's guest is Damian Wilmot, the Chief Legal Officer of BridgeBio Pharma, Inc. This is a packed conversation with tips on how to strategically risk-take to advance your career. Benefit from Damian's expertise in global compliance, crisis management, and employment law, and discover how he built powerful relationships that propelled his professional journey. Don't miss this chance to learn from one of Boston's most influential leaders.Show NotesLinkedIn: https://www.linkedin.com/in/damianwilmot/ Visit Kahila.com to learn more....2024-04-0549 minOn Rare“Accentuate the Positive.” Eric is living with Transthyretin Amyloidosis (ATTR).Eric joins David Rintell, Head of Patient Advocacy at BridgeBio and Mandy Rohrig, Director of Patient Advocacy at BridgeBio Gene Therapy in a two-part conversation to talk about his experience of living with Transthyretin Amyloidosis (ATTR). Eric was an extremely active person who enjoyed sports, riding horses and working on his 20-acre property when he began to have issues which he now attributes to ATTR. Eric’s diagnostic journey took 14 years and as the disease progressed, he experienced two carpal tunnel surgeries, and several other cardiac procedures. In fact, by the time he was diagnosed, Eric could not walk th...2024-04-0333 min
CONKERS' CORNERTWIN PETES INVESTING Podcast no.123: With special guest Algy Hall of Citywire, Finding winning elite companies, Rolls Royce, Unilever, Renault, Reckitt Benckiser, BridgeBio Pharma, Mortgage Advice Bureau, Moneysupermarket, GSK Haleon, Ai, Funds, Stocks &The topics, stocks and shares mentions / discussed include:
Special guest Algy Hall of Citywire
Elite Companies
Top Fund Managers / Top Portfolio Managers
Rolls Royce / RR.
Unilever / ULVR
Renault / RNO
Reckitt Benckiser / RKT
BridgeBio Pharma / BBIO
Mortgage Advice Bureau (Holdings) / MAB1
Moneysupermarket.com / MONY
Thor Industries / THO
Inditex Group / BMAD
Tractor Supply Co. / TSCO
Macerich Co. / MAC
GSK / Haleon / HLN
Biotech
Consumer Staples
Funds
Investment Trusts
Phil Oakley's blog Investingstuff | Phil Oakley | Substack
Harriman House books
Powder Monkey Brewing...2024-03-221h 21On Rare“Having a brother with a rare condition has changed who I am. My family has a big heart because of my brother.”Join David Rintell, Head of Patient Advocacy at BridgeBio and Mandy Rohrig, Director of Patient Advocacy at BridgeBio Gene Therapy as they recognize Rare Disease Day, February 29, 2024, by speaking with siblings of people living with rare conditions. It is often said that a rare condition or disease impacts the entire family, and this is particularly true for rare siblings. Due to the extra care needed by a child living with a rare condition, rare siblings may sometimes miss out on time or attention from their parents. Rare siblings often contribute to caregiving in their families. Though being a rare...2024-02-2645 min
The Latest on the Law: Updates from the Boston BarRecent Delaware Law Updates Affecting M&A TransactionsThis program will cover recent opinions of the Delaware courts on important aspects of M&A agreements, including remedies for damages based on “lost premiums” available to sellers and the parties capable of brining those claims and equitable limits on specific performance, as well as equitable and process considerations, including the standard of review applicable in controller buyouts as well as claims for aiding and abetting breach of fiduciary duty on the part of buyers.
The program will address, among others, the Court of Chancery’s opinion in Crispo v. Musk finally resolving Delaware’s take on so-c...2024-01-3153 minOn Rare“You have to find your people. It’s the most important thing you do along this journey.” Effie is the mother of Ford, a boy living with CTNNB1 syndrome and host of Once Upon a Gene.Becoming a parent to a child with a rare disease was extremely isolating for Effie. In the second episode of our two-part series with Effie, she shares that finding the world of podcasts, documenting similar experiences to her own, was magical. Effie explains how important it is to find others who "get it" and how this feeling of belonging changed everything for her. As Effie explains, “In this community of rare disease families, every story matters, every effort counts, and every heart is connected. We are stronger together and are empowered by the love that guides us.” She describes her...2024-01-2930 minBiotech HangoutEpisode 86This week, hosts Daphne Zohar, Brad Loncar, Josh Schimmer, John Maraganore and Grace Colon discuss the news of the week including several early-stage IPO filings that kicked off the year: Kyverna Therapeutics, CG Oncology and Alto Neuroscience. The hosts also cover AI enabled biomarkers following the FDA’s clearance of two AI diagnostic tools, plus the FDA expands approval of Vertex and CRISPR’s Cagevy as a first CRISPR-edited therapy for beta-thalassemia. They also discuss early-stage funding and an amicus brief that many biotech executives have signed, following the decision on Mifepristone. Other topics include BridgeBio Pharma securing capital from...2024-01-251h 02On Rare“I didn’t know I was part of a rare community until I started listening to the Two Disabled Dudes podcast.” Effie is the mother of Ford, a boy living with CTNNB1 syndrome and host of Once Upon a Gene.Effie Parks, a rare mom, a patient advocate, a podcaster, and host of “Once Upon a Gene” joins David Rintell, Head of Patient Advocacy at BridgeBio to share her story. Recorded during the 2023 Global Genes Conference in San Diego, Effie talks about her journey into motherhood and into the rare community. Effie’s son, Ford, was born with CTNNB1 syndrome, an extremely rare genetic condition. Isolated and trying to navigate her new life as a rare mom, Effie stumbled across Two Disabled Dudes and on first listen, realized she now had a community who understood her experiences. With this new un...2024-01-1923 minOn RareWe listened, we learned, we laughed and we cried! On Rare celebrates the incredible conversations of 2023!In our final episode of 2023, On Rare looks back at the wonderful, rare conversations with our extraordinary guests and David Rintell, Global Head of Patient Advocacy at BridgeBio. Mandy Rohrig, Director of Patient Advocacy at BridgeBio Gene Therapy and host of On Rare, walks us through highlights from the year. We listened, we learned, we laughed and we cried. Join us for a look back at 2023 and don’t forget to subscribe to learn more from our guests in 2024.
2023-12-2244 minOn Rare“In some ways, the sun has been my mortal enemy.” Jay is living with erythropoietic protoporphyria (EPP).Jay joins David Rintell, Head of Patient Advocacy at BridgeBio to talk about his experience of living with erythropoietic protoporphyria (EPP). When Jay was very young, it was difficult to receive a diagnosis for his condition. It took a series of visits to various doctors, including psychiatrists, before a pediatric dermatologist diagnosed him at six years old. The symptoms of EPP are often invisible, which can make diagnosis quite difficult. With vulnerability and clarity, Jay explains that although he may look like everyone else, part of his daily mental energy is utilized managing and planning how to limit his...2023-12-0441 minOn Rare“Improvise. Adapt. Overcome.” Dan is living with limb-girdle muscular dystrophy, type 2I/R9 (LGMD2I/R9).Dan joins David Rintell, Head of Patient Advocacy at BridgeBio to share the story of his long diagnostic odyssey and how ultimately a cancer scare attributed to his LGMD2I/R9 diagnosis. Dan shares that, during this time, he felt relief receiving a diagnosis, but it was quickly paired with the fear of facing the unknown of a progressive neuromuscular disease.
Dan describes the challenges of traveling, living alone and developing the constant fear of falling. Fiercely independent, Dan is concerned about the future. Living with a progressive condition means that you are always forced to adapt a...2023-11-0834 minOn Rare“People with EPP are shadow jumpers.” Brady is living with erythropoietic protoporphyria (EPP).32 seconds. That’s how much time Brady could tolerate exposure to light on his skin before the onset of deep burning, itching pain. Brady is living with erythropoietic protoporphyria, EPP, a rare inherited disorder that causes the skin to become painful when exposed to sunlight. Seconds, minutes, or hours beyond 32 seconds, for Brady, could result in intense, prolonged pain, which would require hours or days in darkness to recover.
Kristen, Brady’s mother, shares with David Rintell, Head of Patient Advocacy at BridgeBio, how Brady was diagnosed, and how he manages the effects of the condition. EPP affe...2023-09-1442 minOn Rare“Life after the transplant wasn’t straightforward.” Anne is living with autosomal dominant polycystic kidney disease (ADPKD)Anne and Mike continue their conversation with David Rintell, Head of Patient Advocacy at BridgeBio, in part two of our autosomal dominant polycystic kidney disease (ADPKD) episode. Anne describes her recovery from the first kidney transplant along with the complications she attributes to her two native kidneys which were not removed during the initial surgery and issues with polycystic liver disease. In the 11 years following, Anne endured an operation to remove her native kidneys, followed by a double organ transplant, giving her both a new liver and kidney. Her first kidney transplant was generously gifted by a friend. However...2023-08-0131 minOn Rare“We named the new kidney - Bean.” Anne is living with autosomal dominant polycystic kidney disease (ADPKD)Anne joins David Rintell, Head of Patient Advocacy at BridgeBio to talk about how autosomal dominant polycystic kidney disease (ADPKD) has impacted her and the lives of her family members. ADPKD weaves through the branches of Anne’s family tree for generations. When Anne was only six years old, her father died at 46 of ADPKD related kidney failure, dialysis, and kidney transplantation. Two of Anne’s five siblings have ADPKD. One had a successful kidney transplant, but another sibling died while on the waiting list for a new kidney. Anne wasn’t surprised by her ADPKD diagnosis at age 25 and wa...2023-06-3033 minOn Rare“Love isn’t love till it’s given away.” Determination has helped Lacey fulfill her dreams, but it hasn’t stopped the progression of LGMD2I/R9.Lacey joins David Rintell, Head of Patient Advocacy at BridgeBio to talk about how LGMD2I/R9 has impacted her life. When Lacey was 16, she was diagnosed with a progressive neuromuscular disease, known as LGMD. The only question she had for the doctor was whether she could one day become a mother. Without additional explanation, she was told that LGMD2I would keep her from having children. For years, Lacey lived in denial. Despite the limitations of her disease, she charged ahead with her life. She runs a business with her husband. She is a wife, mother, and foster...2023-06-0246 minOn Rare“You can brush it off or you can speak up for yourself” Kristen has been advocating for herself and others who live with achondroplasia since she was six years old.Kristen joins David Rintell, Head of Patient Advocacy at BridgeBio to tell her personal story about living with achondroplasia and how she became an advocate at just six years old. By third grade, Kristen was speaking to classrooms about what it is like to live with achondroplasia. In the episode, Kristen shares how she has dealt with health issues, overcame adversity and even published her story in the book, Little Legs, Big Heart. She explains that achondroplasia isn’t just about height – there are other important quality of life concerns which she has learned to address, while also helping othe...2023-04-0745 minOn Rare“When life gives you lemons, make lemon gelato!” Brent is disabled by PKAN, a rare neurodegenerative disease, yet supported by his family he is making many of his dreams come true.Veronica joins David Rintell, Head of Patient Advocacy at BridgeBio to tell her son Brent’s story. When Brent was nine, his family discovered that he had a rare genetic disease called Pantothenate Kinase-Associated Neurodegeneration (PKAN), a condition without any available treatments. Veronica shares how she and her family have supported Brent as he has stubbornly and victoriously succeeded in giving his valedictorian speech in high school, attended community college, and started a local gelato business.
Mallory Harden, Ph.D., Director of Business and Operations at CoA Therapeutics, a BridgeBio company, provides an overview of PKAN...2023-02-2844 minOn Rare“I learned to meditate.” Holly and Beth learned to take care of themselves while supporting their families through their husbands’ ALS diagnoses.In part two of our series on ALS, amyotrophic lateral sclerosis, Holly and Beth, two women whose husbands died from ALS, (also known as Lou Gehrig’s disease) continue their conversation with David Rintell, head of patient advocacy at BridgeBio. They describe the bond they developed over their shared experience and how they continue to offer support to others who have experienced an ALS diagnosis.
Listen to part one to hear Rachel Groth, Ph.D., vice president and head of neuroscience research at BridgeBio, provide an overview of ALS, a fatal neurodegenerative disease that affects nerve cells in...2023-01-1125 minOn Rare“They would go to see their doctors and tell them everything was fine, but everything was not fine.” Carl and Chris struggled in their own ways to accept their ALS diagnosis.Holly and Beth, two women whose husbands died from ALS, amyotrophic lateral sclerosis, (also known as Lou Gehrig’s disease) join David Rintell, head of patient advocacy at BridgeBio to share their experience. ALS is a progressive and fatal neurodegenerative disease that affects nerve cells in the brain and spinal cord.
Rachel Groth, Ph.D., vice president and head of neuroscience research at BridgeBio, provides an overview of ALS. She explains how this devastating disease is characterized by the selective and progressive loss of motor neurons, which are cells in the body and brain controlling mus...2022-12-1539 min
First In Human By VialEpisode 4: Justin To - Chief Operating Officer at BridgeBio PharmaFor Episode 4, we find out the key to unlocking clinical strategy as it pertains to rare diseases with Justin To - Chief Operating Officer at BridgeBio Pharma.To view the full episode transcript, click here.🎧 Stay in the Loop!For the latest news and updates, visit our website: https://vial.comFollow us on social media for real-time insights:Twitter: https://twitter.com/VialTrialsLinkedIn: https://www.linkedin.com/company/vialtrials2022-11-2911 minOn Rare“With CAH, you’re always planning and worried.” Jihan’s two daughters are living with congenital adrenal hyperplasia (CAH).The birth of Jihan’s first daughter, Bella, was traumatic. She and her husband were totally unprepared for the diagnosis of congenital adrenal hyperplasia (CAH) and struggled with the way the news was delivered to them. Their health care providers failed to provide adequate instructions about the care of a baby who is fragile due to adrenal insufficiency. The first two weeks of Bella’s life were frightening, as her parents tried to get the care that she needed and learn how to care for her at home. Twelve years later, Jihan’s youngest daughter, Alessia was also born with C...2022-11-0144 min
矽谷為什麼？EP122 - 生物科技的創新歷程和各階段挑戰 |專訪Octant 策略副總裁 Hannah Shen 沈敬涵沈敬涵 (Hannah Shen) 來自台灣的知名學府，從北一女、台大，一路走到了哈佛，拿到了分子與細胞生物所博士學位，在博士期間，就已經在 Nature 發表了一篇第一作者的文章。 之後眼光獨具的 Hannah，待過了幾間如今估值都一飛沖天的新創企業。第一份工作是在生技軟體公司 Benchling 做業務開發，加入時是公司正處募完A輪，是該公司第十七名員工，如今公司估值美金六十億。 後來 Hannah 待過 BridgeBio Pharma，三年內就從基層的 Associate 升到 VP of Asset Acquisition。帶領五人團隊，尋找、研究以及新創立超過十五個子公司，研發不同的罕見疾病與癌症標靶治療，而公司在這段時間也從 Series B 到 IPO。 如今 Hannah 在生技新創公司 Octant Bio ，擔任策略VP，負責公司的經營與研發策略。平常除了和太太爬爬山、和貓睡覺，也喜歡栽種各種花草。 想知道Hannah如何在海外經營這令人稱羨的職涯嗎？想知道她又看到生技業有甚麼挑戰與機會嗎？歡迎收聽《矽谷為什麼》！ 討論大綱： ✅身為台灣孩子，如何有效率地學習英文？ ✅一路從名校走來，尤其是哈佛，如何改變了你的人生視野？ ✅生物科技產業這幾年有甚麼樣的變化與創新機會？ ✅取得博士學位後，為什麼決定走向商業與策略取向的工作內容？ ✅台灣又該如何接軌美國生科市場與資源 ? -------------------------- 【謝榮雅的想像力商學院】 #商業思維 #設計思考 課程介紹 https://go.sat.cool/4hmwxw 結帳輸入專屬優惠碼 ic500 可以再折 $500 元 2022/10/31 前 #限時優於 43 折 ============ 主持人介紹： 詹益鑑 IC Taiwan Global Angels 創辦人，工程加商管背景。2004-2019 在台灣經歷三次創業、四種產業、五家創投，曾任 AppWorks 合夥人、BioHub Taiwan 助執行長、Startup Genome 台灣新創生態系大使，2020年初旅居矽谷，並於 2021年加入互貴興業擔任策略長，從事數位醫療與生醫產業的投資併購和國際市場開發。 Dr. IC 粉絲頁: https://www.facebook.com/dr.ic.page/ 謝凱婷 KT (矽谷美味人妻) 「矽谷影響力基金會」執行長, 亦是天使投資人, 創業家和料理作家，目前定居在矽谷，社群媒體追蹤數超過100萬人以上。「矽谷影響力基金會」有計畫的推動台灣人在矽谷科技界的活動和新創投資。 以慈善為目的，幫助台灣和矽谷連結，推動更多台灣人在美國的發展。創辦的美味生活HowLiving是全球華人料理家居網站，製作超過三千集的料理影音和直播節目，在華人料理圈有廣大影響力和知名度。曾擔任長榮集團媒體公關，國際企業產品經理，並接受過百家中外國際媒體專訪。 擅長社群行銷、數位內容、KOL孵化，協助許多台灣和美國公司進行數位轉型計畫。 矽谷美味人妻 FB: https://www.facebook.com/kthu1031 美味生活 全球華人料理家居平台 www.how-living.com ============ 歡迎與我們討論 「矽谷為什麼」 FB粉絲頁 bit.ly/39xLlDR 「矽谷創新趨勢交流平台」社團 bit.ly/3oBAFvM ============ 在這邊也能收聽到我們的最新節目唷！ Apple Podcast→ meetstartup.pse.is/QEX8Y Spotify→ meetstartup.pse.is/RFBKL Firstory→ meetstartup.pse.is/3fzp96 SoundOn→ meetstartup.pse.is/QQX2G Youtube→ meetstartup.pse.is/3e6f38 Castbox→ meetstartup.pse.is/QHQL6 ============ 感謝 #國發會 Startup Island TAIWAN 和 @數位時代 @創業小聚 Meet Startup 獨家贊助本節目！ Powered by Firstory Hosting2022-10-2046 minOn Rare“ADH1 doesn’t have me. I have ADH1” Jessica is living with autosomal dominant hypocalcemia type 1 (ADH1).At birth, Jessica’s physician noticed she had low levels of calcium. Genetic testing later confirmed that she had autosomal dominant hypocalcemia type 1 (ADH1) with a calcium receptor (CaSR) gene mutation. Jessica had complications due to ADH1 quite early in life. Before Jessica started kindergarten, she had painfully passed her first kidney stone. Fiercely protective and attentive, Jessica’s mother quit her job to take care of Jessica full-time. She researched and provided Jessica with the latest cutting-edge treatments.
Despite the challenges of living with ADH1, Jessica has done well. She is a hopeful, new mother who is re...2022-10-0533 minOn Rare”He loves curse words! He’s a typical 15-year-old boy who is living with Canavan disease.” Dylan’s parents talk about raising their happy son.
In this month’s episode of On Rare, David Rintell speaks with Cliff and Noreen, the parents of Dylan, a playful, adopted, 15-year-old living with Canavan disease. It’s a heartwarming, funny and poignant conversation about Dylan’s life, the challenges of providing constant care for a severely disabled child and the joy he brings them. As parents of a child with an ultra-rare, fatal genetic condition, Cliff and Noreen live day by day. As Cliff explains, “We focus on what we have now with Dylan and the good times.”
2022-09-0145 minOn Rare”Mom’s just tired. I was in this daze of denial.” A teenager grapples with her mother’s diagnosis of cholangiocarcinoma.In this month’s episode of On Rare, David Rintell speaks with Alex, 18-year-old whose mother is living with cholangiocarcinoma. It’s a moving conversation about a teenager facing the challenges of everyday life while also handling the possibility of life without her mother. We hear what it is like for this young woman to watch her mother struggle to receive a diagnosis, finally learn she has cholangiocarcinoma, and seek treatment options.
2022-07-3127 minOn RareI’m a salt waster, 1 in 20,000. You don’t let it run you, you run it.In this month’s episode of On Rare, David Rintell speaks with Erica, who is living with congenital adrenal hyperplasia (CAH). Erica describes what it has been like to live with CAH and how she decided to manage her condition and live life on her own terms. With humor and honesty, she explains how she’s gone from being a rebellious child to a leader and advocate in the CAH community. Kamal Bharucha, vice president of clinical development at BridgeBio’s affiliate, Adrenas, joins the conversation to provide an overview of the genetic disease and explain how it is diagno...2022-06-3030 minOn Rare“He’s a fighter and takes on whatever is thrown his way.“ Parker is living with tuberous sclerosis complex (TSC).Andrea and Ryan’s son, Parker, is living with tuberous sclerosis complex (TSC). During an ultrasound in the thirty-fifth week of Andrea’s pregnancy, her doctor discovered that Parker had a cluster of tumors in his heart. These tumors, known as rhabdomyoma, were a sign that he might have TSC. Five days after he was born, Parker had heart surgery to remove these tumors and his diagnosis was confirmed. Living with TSC means that Parker and his parents never know when he might have seizures, where new tumors might appear, how these tumors might affect him or what other symp...2022-06-0138 minOn Rare“Hi, I’m nine years old. I have achondroplasia. Would you like to play?” A resilient, athletic child lives with achondroplasiaInês’ 9-year-old daughter, Clara, is living with achondroplasia. In this episode of On Rare, Inês describes what it was like to learn that her daughter has achondroplasia. She describes the steps she took to get Clara the medical care she needed in the early years of her life and how, in the midst of this stress, she remembered to enjoy being a mother to her newborn daughter. Finally, she tells us how she has watched Clara become a resilient, athletic child who has learned to compete against herself in a world that often caters to taller individuals.
Daniela R...2022-04-2941 minOn Rare“I am not going to let cancer stop me.” Driven and determined while living with cholangiocarcinoma
In her 20s, Chaundra believed she was living the dream with a graduate degree and a new, exciting job. Unfortunately, at the age of 29, results from a routine blood test led to a diagnosis of cholangiocarcinoma. Six years later, Chaundra has endured chemotherapy treatments, surgeries, biopsies, multiple clinical trials and much more. Despite her diagnosis, Chaundra is determined and driven. She has taken on new professional opportunities in different cities, has been promoted in her work, fallen in love, and even, run for political office and won.” She tells David Rintell, head of patient advocacy at BridgeBio, “I am...2022-03-3143 minOn Rare“I just want new skin, Mom.” Courage in the face of a painful life with epidermolysis bullosa (EB)Brady is a five-year-old who is living with recessive dystrophic epidermolysis bullosa (RDEB), a rare genetic skin condition. His mother, Eileen, describes what it was like to discover that Brady has RDEB and tells us how she is both the mother of a brave, happy little boy and the nurse who causes him pain during his daily wound care. RDEB has changed their lives.
Sanuj Ravindran, M.D., executive chairman of BridgeBio’s Phoenix Tissue Repair, joins the conversation to talk about the condition and explain how EB affects the body internally and externally.
2022-02-2446 minOn Rare“Something was not right with my muscles.” LGMD2i and the physical struggle to do basic life activitiesJohn and Tony are living with limb girdle muscular dystrophy type 2i (LGMD2i), a rare genetic condition. And while they did not know each other before their joint podcast interview, their stories mirror each other in many ways. They tell us about their challenging and long diagnostic journeys and how LGMD2i has changed the trajectory of their lives and forced them to navigate the world in ways that are often isolating.
Douglas Sproule, M.D., M.Sc., chief medical officer of BridgeBio’s ML Bio Solutions explains how muscles are damaged in people with muscular dystrophy...2022-02-2440 minOn RareBridgeBio Introduces - On RareBridgeBio Pharma introduces On Rare, a podcast about people living with rare conditions.
2022-02-2001 min
Pharmaceutical ExecutiveEpisode 91: A New Way to Partner With AcademiaDr. Michael Henderson, chief business officer of BridgeBio Pharma, and one of Pharm Exec’s 2021 Emerging Pharma Leaders, discusses how his company goes beyond the traditional “hand off” approach between academia and industry, and looks to partner with institutions early on to fill important voids in drug development.2021-09-1618 min
First RoundersNeil KumarNeil Kumar is a co-founder and CEO of BridgeBio Pharma. He discussed with Nature Biotechnology his youth in the Midwest, the founding principles behind BridgeBio, and understanding where your strengths lie. Hosted on Acast. See acast.com/privacy for more information.2021-03-011h 01
Global Venturing Review01 July 2019 – Grab Boosts Series H Round to $4.8bnBig Ones
Southeast Asian ride hailing service Grab has boosted an already swelling series H round to $4.8bn, taking in $300m from investment management firm Invesco.
Deutsche Telekom Capital Partners was launched by its parent company nearly five years ago, and has now formed a $350m second venture and growth capital fund.
Who says the IPO market is on the dip? Adaptive Biotechnologies – backed by Microsoft, Celgene, Illumina, LabCorp and BD Biosciences – saw its share price double on its first day of trading – from $20 to $40.30 – while BridgeBio Pharma, which is developing drugs to treat di...2019-07-0112 minGlobal Venturing Review03 June 2019 – Possible PhonePe Spinout from Flipkart in $1bn DealBig Ones
Right now it feels like we’re gearing up for another period of significant funding, if the rumour mill is anything to go by. Mobile payment platform PhonePe was acquired by Flipkart in 2016, the year after it was founded, but could now be spun back out in a round that could reportedly reach $1bn.
Life insurance firm Northwestern Mutual launched its $50m Northwestern Mutual Future Ventures unit in early 2017 and has since built up one of the more notable CVC portfolios for an insurer, adding the likes of Chime, Ojo Labs and Ladder to...2019-06-0312 min