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Sickle cell disease (SCD) affects 5 million people worldwide, making it one of the most common inherited blood disorders.
Recent breakthroughs in gene therapy have ushered in a new era of hope for SCD patients, offering a potential cure by addressing the genetic root of the disease.
Here is Cellectis’ approach to treat the disease, using TALEN® gene editing tool.
As we move forward, it's essential to continue educating ourselves and others about the potential groundbreaking treatments.
By bridging the knowledge gap, we can ensure that patients and healthcare providers make informed decisions about the future of SCD treatment.
To learn more about the research, follow this link: https://lnkd.in/e68HPhaS
How to treat sickle cell disease with Talent Gene therapy Sickle cell disease, or SCD, is one of the most common inherited disease worldwide. This condition affects 5 million people worldwide and represent a major health public issue that is to be addressed. It is a blood disorder that affects the natural capacity of red blood cells to vectorize oxygen throughout the body. Normal red blood cells are round and flexible to allow them to move easily through blood vessels and deliver oxygen throughout the body. In SCD, or sickle cell disease, the red blood cells are rigid and creation shaped like a sequel, a dysfunctional state that impairs blood flow, oxygen delivery, and so on. It triggers multiple debilitating symptoms including intense pain, crisis, organ damage, strokes and increased risk of infections. We know the origin of this disease. It is caused by a single point typo in patient genome, a mutation in the HBB gene, which encodes the hemoglobin, the enzyme responsible for the oxygen transport throughout the body. The good news is that we know how to correct this typo. Select This developed a clinically relevant gene editing process in HSBC to correct these typo. So how does it work? Well, we have a sentence of DNA that contain one single typo associated to sickle cell disease using our gene editing tool named Talent. The idea is to cut these gene right at the typo and substitute this typo by the correct DNA sentence to eventually correct the dysfunctional gene. This is how we obtain a fully repaired hemoglobin sentence that is now able to produce a functional hemoglobin. This talent gene therapy approach could represent a new alternative treatment especially for patient with limited therapeutic options. This generating process bears a strong therapeutic potential as it could be easily used to correct the typo associated to many other genetic diseases.
General Manager at "Akums Drugs and Pharmaceuticals Limited" CDMO Business | New Buss. Development | In-Out Licensing | Strategic Partnership | Exports - Strong hold on India, Africa, Asia, SEA, Latam and CIS countries
Akums a India's largest CDMO company, having 13 Formulation Plants, 3 API Plants and 4 R&D facilities, catering 13.5% of total medicine consumption of country, have developed a Groundbreaking (First Time in World) Room Temperature Stable Hydroxyurea, Suspension for the management for Sickle Cell Disease (SCD).
Collaborate with us to make the medicine available to the Patients who are suffering with SCD and are in need of affective medicines.
We are quickly approaching an inflection point with respect to using cell therapies to treat #autoimmune disease. While we have increasing confidence that cell therapies will play a pivotal role in the treatment of autoimmune disorders, sponsors will have to overcome significant barriers to deliver on this promise: reducing manufacturing costs, expanding treatment infrastructure, improving the risk/benefit profile, to name just a few.
Curious to learn more? Check out our blog post and presentation at #ISCT2024 by Health Advances Cell and Gene Therapy practice co-leader Ned Wydysh PhD, and Jamie Pierson of Parexel.
Read our blog here: https://bit.ly/3yZzelCISCT, International Society for Cell & Gene Therapy#CGT#CellandGene
Gene therapies for inherited retinal dystrophies give hope to patients and families but developing successful gene therapies and implementing them in clinical practise can be a bumpy ride that requires multidisciplinary collaboration as shown in this new paper by Sarah Wadmann: https://lnkd.in/dAmDFQXm
Cell and gene therapies may have the ability to treat many who have complex diseases. Given the complexity and costs associated with these trials, you need a clinical research partner who can help you navigate these challenges and make sure your trial runs smoothly. Read our white paper to learn more about the importance of strategic alliances needed for cell & gene therapy trials: https://hubs.la/Q02tNsnC0#CellandGeneTherapy#CellTherapy#GeneTherapy#ClinicalTrials#ClinicalResearch
No one wants a world where life-saving treatments are delayed or denied due to red tape.
Cell and gene therapies hold the promise to cure diseases once thought incurable, but without addressing the regulatory challenges, these breakthroughs may never reach those in need.
Join me on Monday, October 7th at 11:30am at #CGMesa24 for a panel discussion on “The Future of Medicine: Approved Cell and Gene Therapies and Breakthroughs on the Horizon.”
We’ll explore:
– the latest advancements,
– the impact of these therapies, and
– how we can overcome regulatory hurdles.
#celltherapy#genetherapy#precisionmedicine | Anshul Mangal
👇 The link to register is in the comments.
How can a collaborative partnership between Veristat and a sponsor of a Phase I/II clinical study for a gene therapy help address critical patient, lab, and manufacturing logistics?
Read our case study to learn how our full service clinical trial team delivered with tight coordination of the treatment’s dosing administration and data review 👇
https://bit.ly/48Mtrw4#fullserviceclinicaltrials#raremetabolicdiseases
We’re only four days away from ARM’s 2024 Congressional Cell & Gene Fly-In to advocate for patient access to cell and gene therapies. Here’s our second ask to Congress.
Ask #2 – Support the Accelerating Kids’ Access to Care Act (H.R. 4758/S. 2372)
Why? It can alleviate burdens for children who need to travel across state lines to receive cell and gene therapies.
#CGFlyIn24
At the end of last year, Vertex/CRISPR and Bluebird received approvals on the same day for two different gene therapy products for sickle cell disease (SCD). Click the link below to download our most recent white paper and explore with our Cell and Gene Therapy experts, Subreen Khatib, Ph.D. and Sarah Jims, the approaches by each company to commercializing these complex technologies in this unique and quite interesting scenario.
https://lnkd.in/eEhVZMjB#cellandgene#CGT
Just concluded, in the Biotech & HealthTech Track, the session, 'Cell and Gene Therapies – The Future is Here', which explored groundbreaking advancements in genetic engineering and personalized medicine. Panelists shared insights into how these cutting-edge therapies are transforming the treatment of previously untreatable diseases, offering new hope for patients worldwide. From gene editing to tailored therapies, this session charted the exciting future of these rapidly evolving fields that are redefining what’s possible in medicine. #BTS2024#BTSUnbound#BreakingBoundaries#BengaluruTechSummit
At Tenaya, we are researching gene therapies for genetic heart conditions, like #ARVC.
Tenaya’s investigational gene therapy is intended to target muscle cells in the heart to treat the underlying genetic cause of ARVC.
Learn more about TN-401 and how it works by watching the full video: https://bit.ly/47YvVIS#GeneTherapy#HeartDisease#Tenaya#ARVC#PKP2
General Manager at "Akums Drugs and Pharmaceuticals Limited" CDMO Business | New Buss. Development | In-Out Licensing | Strategic Partnership | Exports - Strong hold on India, Africa, Asia, SEA, Latam and CIS countries
1moAkums a India's largest CDMO company, having 13 Formulation Plants, 3 API Plants and 4 R&D facilities, catering 13.5% of total medicine consumption of country, have developed a Groundbreaking (First Time in World) Room Temperature Stable Hydroxyurea, Suspension for the management for Sickle Cell Disease (SCD). Collaborate with us to make the medicine available to the Patients who are suffering with SCD and are in need of affective medicines.