Monthly Vibe Bio Newsletter - August 2023
Discover more from Vibe Bio
Monthly Vibe Bio Newsletter - August 2023
Every Cure for Every Community - Progress in the Fight Against Rare Disease
Welcome to ’s Monthly Newsletter! Stay up-to-date with our latest company updates and insights into the ever-evolving biotech funding landscape.
Hi everyone!
In today’s newsletter, we share updates on important rare disease related news and announcements. At the heart of our community lies a shared conviction: to discover cures for every condition. This is a massive undertaking and we realize the challenges we face are great.
Despite the funding challenges in the current economic landscape, we're witnessing significant strides in elevating awareness and comprehension for the rare disease patient community and their loved ones.
As a community, we must continue to be the cheerleaders that policymakers, researchers, investors, and families need to keep pushing advancements forward for future generations.
Join us on Discord and LinkedIn to connect with patients, investors, and researchers in the Vibe Bio community.
Rare Disease Industry Updates
Our partners at Chelsea’s Hope, along with researchers at Institut Químic de Sarrià (IQS) - Ramon Llull University (URL) in Barcelona released a eye-opening documentary called Fighting the Rare. Fighting the Rare is a glimpse into research on Lafora disease, a rare neurodegenerative condition, through the testimonies of researchers, patients and their relatives.
AstraZeneca's Rare Disease Arm in $1 Billion Deal for Pfizer Gene Therapies
Delaware Enacts Law to Address Needs of Rare Disease Patients and Families
Senate Bill 55 (SB 55) with Amendment SA 1, which officially establishes a Rare Disease Advisory Council (RDAC) in their state.
FDA Accepts Nintedanib sNDA for Pediatric Fibrosing Interstitial Lung Disease
HEC Forum: Understanding Rare Disease Experiences Through the Concept of Morally Problematic Situations
“The objectives of this study were to:
(1) acquire an in-depth understanding of morally problematic situations experienced by adults living with rare diseases in the province of Québec and
(2) to develop an integrative model of the concept of morally problematic situations. To this end, an online survey targeting this population was developed through a participatory action research project.”
Study sheds light on mechanisms behind a rare disease found almost exclusively in boys
New method used to develop RNA therapy for the treatment of rare diseases
“The research is based on splice-switching antisense oligonucleotides (ASOs). ASOs are short sequences of synthetic nucleic acids, a chain of nucleotides adenine (A), cytosine (C), guanine (G) and thymine (T), connected in a specific sequence. The specific sequence is called "antisense" because it complements an RNA target sequence, binding with it to alter its function.”
We want to hear from you!
If you enjoyed this update or have any others you’d like to share, let us know in the comments below!
Which rare disease companies should we be following? Are you a biotech founder working to solve some of the biggest challenges in understanding and treating rare diseases? We would love to support you.
Learn about Inflection Point Financing (IPF) 💰
Vibe Bio’s strategy supports biotechs through Inflection Point Financing (IPF).
Utilizing our extensive network of industry experts, Vibe Bio expeditiously assesses high-potential drug programs, unlocking capital through a deferred payment arrangement.
IPF enables biotech companies to dedicate more time on science and less time fundraising, marking a shift in the biotech fundraising paradigm and prevents potentially groundbreaking research from being abandoned.
💰 If you are seeking novel and rapid financing strategies to drive you towards your next inflection point, Vibe Bio would like to speak with you. Let’s explore opportunities together.
VibeCast Updates 🎙️
On VibeCast, our Industry Engagement Manager and show host Ray Dogum, interviews Vibe Bio community experts who come from diverse backgrounds and career paths. However, one commonality that all VibeCast guests have is a passion to improve society by encouraging research and educating others by sharing their own experiences.
Collectively we have the skills. We have the technology. We have the passion. We now need the community catalyst to bring it all together. That’s Vibe.
In addition to our YouTube channel, you can listen to episodes on:
We are always looking for interesting and knowledgeable people to interview on our show. If you know of a community member who would be a great guest, please let us know. You can suggest a guest by sending us an email, leaving a Substack comment below, or reaching out via social media.
Check out the latest episodes 19-22 below.
Lafora Disease Experts on Clinical Trials, Challenges Funding Rare Disease Research | VibeCast Ep 19
Niki Markou lives in Sydney, Australia and is the mother of a 19 year old girl named Angelina who has Lafora disease. She volunteers on the board of Chelsea’s Hope Lafora Children Research Fund. Previous to her daughter's diagnosis, she had a full time career in marketing. Niki joined the board in 2021, starting off as a parent advocate to help families just like her stay informed and where she still continues to be in close contact with many families around the world. Her main role has been creating all marketing content and liaise with fellow board members, researchers, doctors and organizations to create more awareness for Lafora disease and find possible treatments that could lead to a cure.
Jordi Duran is a Principal Investigator and Associate Professor at Institut Químic de Sarrià (IQS) - Ramon Llull University (URL) in Barcelona. His research group is devoted to the study of brain metabolism, in particular the physiological roles of glycogen in brain functioning and its pathological role in neurodegenerative conditions like Lafora disease.
Watch their documentary, Fighting the Rare, for a deeper dive into Lafora disease and how it impacts patients and families.
Ethan Perlstein on the Role of Families in Research Funding and 1-to-N Medicine | VibeCast Ep 20
Ethan O. Perlstein received a PhD in 2006 from Harvard University (Department of Molecular and Cell Biology) while working in the laboratory of Professor Stuart Schreiber. He completed an independent postdoctoral fellowship at the Lewis-Sigler Institute at Princeton University from 2007 to 2012. Since its founding in 2014, he is CEO of Perlara PBC, the first biotech Public Benefit Corporation partnering with highly motivated families to develop treatments and cures for genetic diseases. Perlara’s first joint venture Maggie’s Pearl is the commercial sponsor of a pivotal Phase 3 trial of epalrestat for the treatment of PMM2-CDG.
Stevie Cline on Biotech Startup Investments & Investing in Regulated Spaces | VibeCast Episode 21
Stevie Cline, J.D., L.L.M., Ph.D, is the Managing Director of Vol. 1 Ventures, an early stage venture fund focused on investing in highly regulated industries. Stevie is also the former co-founder of Prenome, a genetics-based healthcare startup focused on predicting and preventing pregnancy complications using custom diagnostic tests, AI, & machine learning. Prior to entering the tech sector, she was a well-known attorney in the M&A and Taxation space. She has also previously held leadership positions at Johns Hopkins University and the American College of Obstetricians & Gynecologists. Stevie is also the founder of
, a newsletter on the regulatory and compliance landscape that has over 20,000 subscribers.Michelle C. Werner on Alltrna's tRNA Medicine for Stop Codon Disease | VibeCast Episode 22
Michelle C. Werner is a seasoned pharmaceutical executive with more than 20 years in the industry spanning both commercial and research & development (R&D) responsibilities. Prior to Alltrna, Michelle served as Worldwide Franchise Head, Solid Tumors at Novartis Oncology, where she was responsible for delivering the disease area strategies across multiple tumors and led business development efforts resulting in a doubling of long-term portfolio value for the franchise. Previous to Novartis, Michelle was a senior leader at AstraZeneca and as Global Franchise Head in Hematology, she was critical in launching multiple indications worldwide for CALQUENCE®. She is currently serving a Board appointment for the non-profit organization Rare Disease Renegades, a purpose that fuels her passions both personally and professionally.
Upcoming Event 📅
September 19th, 6-9pm: Vibe Bio - Meet and Greet Networking Event - Boston, MA
Registration Page with details coming soon. Mark your calendars for this great opportunity to mingle with biotech leaders and investors during Biotech Week Boston.
Community Feedback 📣
We encourage you to explore our Discord server and learn more about our mission to accelerate the development of new treatments and cures through inflection point financing. You can also follow us on social media to stay up-to-date with the latest news and updates from our community.
Tell us what else you’d like to see in future editions of the newsletter. We’d love to hear from you!
Sincerely,
The Vibe Bio Community Team
#EveryCureforEveryCommunity