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Alliance for Regenerative Medicine
Registrace 8. 11. 2012
The Alliance for Regenerative Medicine (ARM) is the leading international advocacy organization dedicated to realizing the promise of regenerative medicines and advanced therapies. ARM promotes legislative, regulatory and reimbursement initiatives to advance this innovative and transformative sector, which includes cell therapies, gene therapies and tissue-based therapies. Early products to market have demonstrated profound, durable and potentially curative benefits that are already helping thousands of patients worldwide, many of whom have no other viable treatment options. Hundreds of additional product candidates contribute to a robust pipeline of potentially life-changing regenerative medicines and advanced therapies. In its 11-year history, ARM has become the voice of the sector, representing the interests of 350+ members worldwide, including small and large companies, academic research institutions, major medical centers and patient groups.
OTP Update
This webinar features Dr. Nicole Verdun, Super Office Director of the Office of Therapeutic Products (OTP) at CBER and Dr. Rachael Anatol, Deputy Super Office Director of OTP. Dr. Verdun will provide an update on the status of the new OTP Super Office, including hiring progress and an overview of the organization as it currently stands.
zhlédnutí: 277
Video
CAR-T: A DEMI-DECADE IN THE REAL WORLD
zhlédnutí 189Před měsícem
This panel will reflect on the remarkable journey of CAR-T therapies over the past five years, exploring their impact on patients in real-world scenarios. Our expert panelists will highlight clinical data and share positive outcomes observed with commercial CAR-T products. Discover the tangible benefits these therapies have brought to the broader patient community thus far and gain valuable ins...
INNOVATIVE PAYMENT SOLUTIONS - RECENT LEARNINGS FROM GENE THERAPY LAUNCHES
zhlédnutí 55Před měsícem
Attendees will gain valuable insights into the latest strategies employed in successfully introducing gene therapies to the market, including innovative payment models and emerging best practices. The discussion will not only spotlight recent successes but also explore the pursuit of sustainability and the development of new models that shape the future of gene therapy launches. Moderator: Paol...
THE EU JOINT HEALTH TECHNOLOGY ASSESSMENT: A FIT FOR PURPOSE JCA?
zhlédnutí 50Před měsícem
This panel will delve into the anticipated impact of the JCA on the ATMP sector in Europe in 2025 and beyond. Speakers will examine strategies for crafting compelling evidence for regulators and HTAs, assess if the European HTA coordination is going to simplify or complicate things, and scrutinize whether national Pricing and Reimbursement bodies will effectively access the necessary informatio...
WELCOME REMARKS
zhlédnutí 59Před měsícem
Speakers: Tim Hunt, CEO, Alliance for Regenerative Medicine (ARM) Devyn Smith, Ph.D., CEO, Arbor Biotechnologies; Chairman, Alliance for Regenerative Medicine (ARM)
THE FUTURE OF GENETIC MEDICINE
zhlédnutí 337Před měsícem
Driven by technological and scientific advances, the landscape of genetic medicine is rapidly changing. This session will discuss the current state, future technologies and innovations, and solutions for manufacturing and regulatory bottlenecks as new genetic medicines move towards the clinic. Moderator: Nina Bauer, Ph.D., Chief Business Officer, SmartCella Speakers: Behnam Baghbaderani, Ph.D.,...
A FORECAST ON THE 2024 CGTX LANDSCAPE
zhlédnutí 106Před měsícem
Moderator: Anshul Mangal, President, Project Farma and Precision ADVANCE Speakers: Igor Bilinsky, Ph.D., Chief Operating Officer, Iovance Biotherapeutics Phil Cyr, Senior Vice President, Precision Value & Health Doug Danison, Head of Commercial, Cell and Gene Therapy Unit, Bayer Stefan Irion, M.D., Chief Scientific Officer, BlueRock Therapeutics Helen Sabzevari, Ph.D., President and CEO, Precigen
ARM STRATEGIC INITIATIVES
zhlédnutí 21Před měsícem
Speaker: Rita Johnson-Greene, Chief Operating Officer, Alliance for Regenerative Medicine (ARM)
REDEFINING THE GLOBAL REGULATORY LANDSCAPE FOR ATMP COMMERCIAL APPROVAL: 1 APPROVAL TO RULE THEM ALL
zhlédnutí 31Před měsícem
Moderator: Matthew Hewitt, Ph.D., Vice President, Technical Officer CGT and Biologics, Charles River Laboratories Speakers: Jean-Philippe Combal, Pharm.D., Ph.D., Co-founder and CEO, Vivet Therapeutics Mike Lehmicke, Senior Vice President , Science and Industry Affairs, Alliance for Regenerative Medicine (ARM) Devyn Smith, Ph.D., CEO, Arbor Biotechnologies; Chairman, Alliance for Regenerative M...
TISSUE REPAIR AND REGENERATION - REFLECTING ON PROGRESS FOR NOVEL TISSUE THERAPEUTICS
zhlédnutí 65Před měsícem
Moderator: Jeff Ross, Ph.D., President, Miromatrix a United Therapeutics Company Speakers: Petter Björquist, Ph.D., CEO, VERIGRAFT Don Fink, Ph.D., Master Practice Expert, Regulatory, Dark Horse Consulting Tom Lowery, Ph.D., President and Chief Technology Officer, Satellite Bio
FIRESIDE CHAT: THE PATIENT ACCESS ENVIRONMENT FOR ATMPS IN EUROPE - THE ROLE OF EVIDENCE GENERATION
zhlédnutí 29Před měsícem
Moderator: Mark Battaglini, Chief Strategy Officer, Alliance for Regenerative Medicine (ARM) Speaker: Loris Brunetta, Founding Board Member, Thalassaemia International Federation (TIF)
TRANSLATING EARLY RESEARCH INTO PROMISING NEW ATMPS FOR PATIENTS THROUGH PUBLIC-PRIVATE PARTNERSHIPS
zhlédnutí 17Před měsícem
This session will shine the spotlight on European public-private partnership initiatives for ATMPs and why they can play an important role in accelerating patient access to innovative therapies. Moderator: Petter Björquist, Ph.D., CEO, VERIGRAFT Speakers: Elsa Abranches, Ph.D., Senior Director, Head of Cell Therapy Bioprocess Development and Manufacturing, BioPharmaceuticals R&D, AstraZeneca El...
DEVELOPING THE ATMP ECOSYSTEM OF THE FUTURE
zhlédnutí 39Před měsícem
Join our discussion on overcoming regulatory and policy challenges at both European and national levels, as industry experts share insights on fortifying the foundation of ATMPs in the European landscape. Moderator: Nitin Mahadev, Global Head of Gene Therapy Marketing, Pfizer Speakers: Barbara D’Alessio, Board Member, UNIAMO Federazione Italiana Malattie Rare Jim Lund, Ph.D., Chief Business Dev...
FEATURED KEYNOTE SPEAKER: DR. MASSIMO DOMINICI
zhlédnutí 42Před měsícem
FEATURED KEYNOTE SPEAKER: DR. MASSIMO DOMINICI
STRATEGIC INVESTMENT PERSPECTIVES IN THE EVOLVING EUROPEAN AND GLOBAL ATMP LANDSCAPE
zhlédnutí 63Před měsícem
STRATEGIC INVESTMENT PERSPECTIVES IN THE EVOLVING EUROPEAN AND GLOBAL ATMP LANDSCAPE
INNOVATIVE APPROACHES: DECENTRALIZED MANUFACTURING OF ATMPS
zhlédnutí 114Před měsícem
INNOVATIVE APPROACHES: DECENTRALIZED MANUFACTURING OF ATMPS
CTI Clinical Trial & Consulting Services
zhlédnutí 10Před měsícem
CTI Clinical Trial & Consulting Services
good stock to invest in. wish I knew about Krystal in 2018
My husband suffering from cone destrophy please help me out
Hi what is the treatment for cone destrophy we are from India
💉💀
how about producing some collagen-I and collagen-III, the lack of which will cause skin wrinkles.
I have been attending or watching presentations on retinitis pigmentosa for approximately 15 years. I am dismayed by the slowness of the progress. Hopefully, researchers will feel a sense of urgency with this research. As noted in this presentation, there is a point in the progression of RP beyond which this technique does not work. The dreams, hopes, and lives of many persons depend on rapid research success.
I hope they collaborate
amazing talk thank you
Where are they getting skin, blood, and ogans from? And Chimeras?
Go go go! Milk that beauty industry to fund research for rare diseases!
I am Praying every day!! It is so Painful a Life with hearing loss, hyperacusis, Tinnitus And Pain! Please help us. Please ❤️🙏🏼
When is this available in Mexico?
Will it be possible to replace dead tissue that was removed in a debridement from a pilonidal cyst ??? 😢😢😢😢😢😢 The removal of the deeper tissue maybe fascia has really thrown off my balance and causes pain in hip and knee Etc etc
As a liver patient, this presentation was greatly valuable. I wish we could get a more accurate timeline though! Millions of patients are counting on this macrophage therapy!
Sounds great but its disappointing to know that the cell pouch Sernova will use is made of woven polypropylene mesh which is known to cause severe long term side effects. This is seen in hiatal hernia post op surgery patients using this same material. So while someones type 1 maybe cured they could be left with something just as harmful in the long run. One step forward two steps back 🤷♂.
Where can these treatment?
at the end of the presentation he says they are preparing for a phase 3. so unfortunately at least another 2 years. thanks, fda!
🤣
All of my money gone. $2000 stock bought a couple years ago, now after the reverse splits, and delisting from the main exchanges, the stock is worth nothing. I am literally closing an account with one broker I use and just abandoning the stock if they will let me, because the transaction fees to sell it are now more than I would get for selling it ($0.04).
Wow. Amazing
IMPORTANT 14 MIN
Frank Reynolds was ousted from In Vivo Therapeutics in August 2013 for using company funds for his own personal use and for being in general a tyrant and bully to staff (and don't listen to him baloney about being paralyzed and willing himself to walk again--it was all a lie). He subsequently established another company (Pixar Bio) to develop a pain medication delivery platform. Unfortunately, Reynolds was ultimately convicted of securities fraud in 2021 and sentenced to 7 years in prison (where he died in January 2022). The "illustrious" Robert Langer took part in promotional videos for Reynolds scam company (PixarBio) which made him just as culpable in all of this. And as for In Vivo Therapeutics their scaffold failed miserably in clinical trials and the company filed for Chapter 11 bankruptcy in February 2024. I was an investor in In Vivo and was not made aware of Reynolds' shenanigans and I have contacted the SEC to see what can be done. Otherwise, I will have lost all of my investment in this garbage.
This is super exciting stuff! Wishing them the best and hoping they make hair loss a problem of the past!
My mother is on there!
Have you started testing?
I’m cheering for this company and pray that they succeed. My family member has systemic scleroderma with lung involvement and I worry about a future where they are gone.
Keep up this amazing work. Pray for your safety, and success. So many lives depend on this.
Hopefully its a success!
Got fda approval for phase 3 trial 🔥
@@Babayagaom its fantastic news tbh.
আপনারা গরীব যদি দেশকে উদ্ধার করেন তাড়াতাড়ি মানুষের এই চিকিৎসা পাচ্ছে না তাই আপনাদের সহায়তা
Die Möglichkeit Parkinson zu heilen. Bis heute kann man nur die Symptome mehr recht oder schlecht beeinflussen.
সারা পৃথিবী এই ওষুধের জন্য মনে হয় দু-তিন কোটি 4-5 কোটি মানুষ মারা যেতে পারে
Congratulations!
সারাবিশ্বের মধ্যে আপনার মত ভালো মানুষ নাই আপনারা তাড়াতাড়ি মানুষকে বাঁচান জিন গরিব দেশগুলো অসহায় এর মধ্যে সবাই চা জিন
সারা বিশ্বের আপনার কাছে সবাই দোয়া রইল আপনারাই হেতি দই দয়া করে মানুষকে তাড়াতাড়ি জিন থেরাপি
Huge news will be fantastic when/if this comes to fruition
Stem cells from CCR5Δ32 gene mutation from collected samples modified to be universally adaptable CCR5Δ32 stem cells, combined with Excision Bio-Therapeutics' pending HIV functional/sterilizing therapeutic cure, EBT-101 would mostly equate to a sterilizing HIV cure and an eradication of the virus in the human body. [1]. *Excision BioTherapeutics' EBT-101* The pending HIV functional/sterilizing therapy, *EBT-101, developed by Excision BioTherapeutics. Stated by Excision BioTherapeutics' CEO is Daniel Dornbusch, EBT-101 has the potential to eliminate HIV, and EBT-101 is currently in human clinical trials. In September 2022, Excision BioTherapeutics dosed their first participant in the EBT-101 Phase 1/2 Trial. EBT-101 is CRISPR-based, single administered therapy, designed to cut and removing HIV DNA out of human cells. This potential gene editing treatment employs an adeno-associated virus to deliver CRISPR-CAS9 nucleases and dual guide RNAs targeting three sites within the HIV genome that tell the enzymes where to cut. EBT-101 was initially developed by Dr. Kamel Khalili and researchers at Temple University, located in Philadelphia, Pennsylvania. Excision BioTherapeutics received an FDA Fast Track Designation, for EBT-101. The Fast Track Designation is a process designed to facilitate the development, and expedite the review of drugs to treat serious conditions and fill an unmet medical need. This process is designed to expedite the development and review of drugs which may demonstrate substantial improvement over available therapy. With the FDA granting Excision BioTherapeutics a Fast Track Designation for EBT-101. Hopefully, EBT-101 will be available for distribution in a couple of years, as the first three patients were infected with EBT-101 that seeks and destroys lingering pieces of HIV in the body. The first EBT-101 human clinical Phase I/II trial (NCTO5144386) started last year in September 2022. At a recent November 20233 presentation in Brussels, Professor Rachel Presti of Washington University St. Louis School of Medicine reported that all three participants in the first dose cohort, regarding EBT-101, have now received HIV therepy. The initial results indicated that no serious adverse events ir dose-limiting toxicities. There were four mild adverse events that nay have been related to the gene therapy, however all were resolved. Two participants experienced transient liver enzymes elevations. None of the three withdraw from the study. EBT-101 was detectable in the blood of all participants four weeks after receiving a single IV infusion using the first dose level to be evaluated. There was no evidence of "horizontal transmission of gene vector shedding of EBT-101 in two tissue compartments associated with male reproductive function", suggesting the therepy is not passed on in semen, according to Excision BioTherapeutics. These findings support testing of a higher dose of EBT-101 in a second cohort of six people, which will happen this year. Dr. William Kennedy, Excision BioTherapeutics' Senior Vice President of Clinical Development stated in a press release the " Establishing the safety and bio-distribution of EBT-101 is an important first step in the clinical program. These initial observations provide important clinical data that support the advancement of the EBT-101-001 trial to the next dosing cohort". The study participants in the EBT-101-001 trial will be followed fir 48 weeks after EBT-101 us administration, and those who are eligible will be assessed for sustained viral suppression after stopping antiretrovirals in an analytical treatment interruption starting at week 12. The first participant, who received EBT-101 in the sumner of 2022, is already well past the 12-week mark, but Excision BioTherapeutics has not provided any further information about his status. [2]. *Modified CCR5Δ32 Stem Cell Infusion* Currently, bone marrow transplant from CCR5Δ32 mutated cells is a procedure that infuses healthy blood-forming stem cells wihich an HIV-positive subject receives a bone marrow transplant from compatible donor who us homozygous for mutations in the HIV receptor CCR5 that result in resistance to HIV infection. Stem Cells Transplant procedures from donors with CCR5Δ32 cell mutation has been documented to have medically cured numerous individuals with HIV, although this procedure comes with with possible risk. Patients receiving a stem cell from a doner with a *CCR5Δ32 mutation* , which blocks HIV from infecting the body. With these stem cell bone marrow transplants, involving the CCR5Δ32 mutation from a donor is a complicated procedure, normally reserved for cancer patients, and comes with many risks, and is too risky to offer it as a option as an HIV cure for everyone. Some individuals were born with the CCR5Δ32 mutation, which naturally blocks HIV, there were those that are considered LTP (Long-term Progressors), which they will carry HIV for decades before their HIV progress to AIDS (with HIV medications). As of 2023, there have been over five documented cases of HIV positive individuals be totally cured from HIV after completing this procedure.
I have al amyloidosis and multiple Myeloma. Is there something coming up to remove amyloid proteins from heart, kidneys and other organs? How about multiple Myeloma. I would also be interested in clinical trials.
Need this for stargardt too!
Well presented. Steve really resisted the urge to talk too fast while still managing to get all the points across.
Thank❤you
Always amazing to come across a video like this with an expert like Peter - and it has less than 1k views. Thanks for the awesome content!
Can you share the name of the book?
Buenas tardes, escribo desde Uruguay, queremos saber si el webinar anunciado para el 1ro de noviembre por parte del laboratorio Krystal biotecnología, para ampliar información sobre la terapia, lo podrían grabar y subir a CZcams con posibilidad de traducción al español. Es fundamental tener más información, sobre todo nos interesa saber si Vijuvek es aplicable a pacientes con otras formas o mutaciones de la enfermedad, por ejemplo Simplex. Muchas gracias Saludos.
My daughter been on the list for a year now and the person that is in charge of recruiting in houston for Gene therapy for rett I can't even get a hold of any one at all I been calling there is 5 spots available and the doctors can't even call us and talk to us at all why is it so Hurd for the neurologist to call and see where we stand on recruiting our children they just ignore me not calling back and not giving me any info Just sad 😔 We believe in doctors but they take our kids as jokes and it hurts how simply they don't care as simple as a phone call
Can you please look into regeneration medicine for meibomian gland dysfunction?
Can you please look into regeneration medicine for meibomian gland dysfunction?
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On investors - Watch out for those wolves in sheep clothing - The Big Pharma - surely find some reps in the audience !!
Will you be adding stemsons presentation
You got that right!!!!!!!
Does EBT-101 is a drug for hiv sterilizing cure ?