OTP Update
58:01
4 ай бұрын
BlueRock Therapeutics
15:08
4 ай бұрын
Satellite Biosciences
12:35
4 ай бұрын
WELCOME REMARKS
18:55
4 ай бұрын
THE FUTURE OF GENETIC MEDICINE
57:41
ARM STRATEGIC INITIATIVES
14:37
4 ай бұрын
ETHICS IN GENE AND CELL THERAPY
59:24
PolTREG
17:52
4 ай бұрын
MaxCyte
14:42
4 ай бұрын
Cellistic
15:20
4 ай бұрын
GeneFab
0:23
4 ай бұрын
Forge Biologics
13:20
4 ай бұрын
Immpact Bio
15:44
4 ай бұрын
L7 Informatics
14:46
4 ай бұрын
SmartCella
14:37
4 ай бұрын
Пікірлер
@ElijahJihadDay
@ElijahJihadDay 4 күн бұрын
Love the work
@tadleyironwood7595
@tadleyironwood7595 8 күн бұрын
Happy to finally see a promising treatment for RP coming towards the clinic.lets go BlueRock'
@prakashaero1987
@prakashaero1987 12 күн бұрын
Same old story for last 4 years.
@prakashaero1987
@prakashaero1987 12 күн бұрын
It's a bioshell. Another cheat biotech. See where its stock price is. Don't invest in this garbage.
@Mr.Majestic77
@Mr.Majestic77 16 күн бұрын
So this guy still hasn't publicly came out to acknowledge that Excision BioTherapeutics EBT-101 was a complete failure in terms of curing HIV or even slowing viral replication.
@kamonbruhh
@kamonbruhh 23 күн бұрын
When will this happen?
@NaveedSiddiq-e7h
@NaveedSiddiq-e7h Ай бұрын
Anyone know what are the updates on this? A large community having cone rod dystrophy is waiting for the outcome.
@allopatricape
@allopatricape 2 ай бұрын
I think there was an error in the upload, it ends after 22seconds
@DEEPAKKUMAR-xq7vb
@DEEPAKKUMAR-xq7vb 2 ай бұрын
Please do it fast
@bufnyfan1
@bufnyfan1 2 ай бұрын
This company is now "KAPUT". Let the lawsuits begin
@lisagagne3760
@lisagagne3760 3 ай бұрын
Yaaayyy🎉
@harismajeed5144
@harismajeed5144 3 ай бұрын
Will you make hair loss problem solution in this generation 😂
@THEROCKHISTORIAN
@THEROCKHISTORIAN 3 ай бұрын
good stock to invest in. wish I knew about Krystal in 2018
@sandhyav8131
@sandhyav8131 3 ай бұрын
My husband suffering from cone destrophy please help me out
@sandhyav8131
@sandhyav8131 3 ай бұрын
Hi what is the treatment for cone destrophy we are from India
@thedoomsdayman66666
@thedoomsdayman66666 4 ай бұрын
💉💀
@paulfoote2706
@paulfoote2706 4 ай бұрын
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.
@jiezstv2040
@jiezstv2040 25 күн бұрын
Nanoscope therapeutics is seeking approval
@ai-ur5uv
@ai-ur5uv 4 ай бұрын
I hope they collaborate
@ai-ur5uv
@ai-ur5uv 4 ай бұрын
amazing talk thank you
@Twilightsummerbreeze
@Twilightsummerbreeze 4 ай бұрын
Where are they getting skin, blood, and ogans from? And Chimeras?
@expression3639
@expression3639 4 ай бұрын
Go go go! Milk that beauty industry to fund research for rare diseases!
@naelbeno6249
@naelbeno6249 5 ай бұрын
I am Praying every day!! It is so Painful a Life with hearing loss, hyperacusis, Tinnitus And Pain! Please help us. Please ❤️🙏🏼
@xyz--
@xyz-- 5 ай бұрын
When is this available in Mexico?
@blueresonantmonkey3188
@blueresonantmonkey3188 5 ай бұрын
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
@WellHellooooThere
@WellHellooooThere 5 ай бұрын
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!
@KM-vc7iy
@KM-vc7iy 6 ай бұрын
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 🤷‍♂.
@wiroku212
@wiroku212 6 ай бұрын
Where can these treatment?
@reluctantrealist6861
@reluctantrealist6861 5 ай бұрын
at the end of the presentation he says they are preparing for a phase 3. so unfortunately at least another 2 years. thanks, fda!
@rickybp2259
@rickybp2259 3 күн бұрын
@@reluctantrealist6861any alternative elsewhere? Japan, south korea , china? Have they found any alternatives? Any idea?
@viewer5891
@viewer5891 6 ай бұрын
🤣
@daniellesnelleman2022
@daniellesnelleman2022 7 ай бұрын
Wow. Amazing
@DaveGreen-u9b
@DaveGreen-u9b 8 ай бұрын
IMPORTANT 14 MIN
@bufnyfan1
@bufnyfan1 8 ай бұрын
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.
@venom_lowrider
@venom_lowrider 8 ай бұрын
This is super exciting stuff! Wishing them the best and hoping they make hair loss a problem of the past!
@ruiiheffron
@ruiiheffron 8 ай бұрын
My mother is on there!
@theswd7062
@theswd7062 9 ай бұрын
Have you started testing?
@Corrupttt
@Corrupttt 9 ай бұрын
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.
@user-DVpazyamor
@user-DVpazyamor 18 күн бұрын
Praying along with you, hopefully my daughter can be approved for this treatment and help her soon!!
@craigostler8988
@craigostler8988 9 ай бұрын
Keep up this amazing work. Pray for your safety, and success. So many lives depend on this.
@delaware12pwner
@delaware12pwner 9 ай бұрын
Hopefully its a success!
@Babayagaom
@Babayagaom 3 ай бұрын
Got fda approval for phase 3 trial 🔥
@delaware12pwner
@delaware12pwner 3 ай бұрын
@@Babayagaom its fantastic news tbh.
@peny1981
@peny1981 2 ай бұрын
So, at least 3 years. And for the cervical version, you will have to wait even longer.
@Md.MonirIslam-e4j
@Md.MonirIslam-e4j 9 ай бұрын
আপনারা গরীব যদি দেশকে উদ্ধার করেন তাড়াতাড়ি মানুষের এই চিকিৎসা পাচ্ছে না তাই আপনাদের সহায়তা
@guebei1
@guebei1 9 ай бұрын
Die Möglichkeit Parkinson zu heilen. Bis heute kann man nur die Symptome mehr recht oder schlecht beeinflussen.
@Md.MonirIslam-e4j
@Md.MonirIslam-e4j 10 ай бұрын
সারা পৃথিবী এই ওষুধের জন্য মনে হয় দু-তিন কোটি 4-5 কোটি মানুষ মারা যেতে পারে
@dianaiororbia-knapton7679
@dianaiororbia-knapton7679 10 ай бұрын
Congratulations!
@Md.MonirIslam-e4j
@Md.MonirIslam-e4j 10 ай бұрын
সারাবিশ্বের মধ্যে আপনার মত ভালো মানুষ নাই আপনারা তাড়াতাড়ি মানুষকে বাঁচান জিন গরিব দেশগুলো অসহায় এর মধ্যে সবাই চা জিন
@Md.MonirIslam-e4j
@Md.MonirIslam-e4j 10 ай бұрын
সারা বিশ্বের আপনার কাছে সবাই দোয়া রইল আপনারাই হেতি দই দয়া করে মানুষকে তাড়াতাড়ি জিন থেরাপি
@ClockinLoot
@ClockinLoot 10 ай бұрын
Huge news will be fantastic when/if this comes to fruition
@SefsonGH
@SefsonGH 3 ай бұрын
Never happening now
@nohaalmutlaq2007
@nohaalmutlaq2007 20 күн бұрын
@@SefsonGH Why not?
@SefsonGH
@SefsonGH 19 күн бұрын
@@nohaalmutlaq2007 cos they'll never be a cure for it
@Mr.Majestic77
@Mr.Majestic77 10 ай бұрын
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.
@2010twix1
@2010twix1 10 ай бұрын
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.
@jiezstv2040
@jiezstv2040 10 ай бұрын
Need this for stargardt too!
@Tobacc0
@Tobacc0 10 ай бұрын
Well presented. Steve really resisted the urge to talk too fast while still managing to get all the points across.
@shanewest7354
@shanewest7354 10 ай бұрын
Thank❤you