"It is increasingly necessary for developers to explore alternative, non-viral approaches that can enhance the safety and efficacy of these groundbreaking therapies." James Brady, MaxCyte Senior Vice President, Technical Applications and Customer Support, highlights the risks associated with viral vectors in cell and gene therapy (CGT). With safety concerns like oncogenesis and high manufacturing costs, the need for safer alternatives is clear. #Electroporation stands out as a promising non-viral method, offering lower safety risks, greater payload flexibility, and reduced production complexities. This technique is already making waves, as seen with Vertex's FDA-approved CRISPR therapy, Casgevy. Embracing electroporation could revolutionize CGT development. Read more in Genetic Engineering & Biotechnology News: https://lnkd.in/e8jcCEdm
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SalioGen is in the News: Check out this feature article in GEN with Jason Cole, CEO, discussing our Gene Coding technology, ASGCT data presentations, and more.
ASGCT 2024: SalioGen’s Nonviral In Vivo Gene Insertion Technology Takes Preclinical Strides Jason Cole, CEO and Chair of Board of Directors at SalioGen Therapeutics is showing some of the company's first work at the American Society of Gene & Cell Therapy meeting (#ASGCT2024) that shows how their nonviral in vivo gene insertion technology can be used. This technology uses a bioengineered mammalian transposase to integrate large DNA constructs permanently.
ASGCT 2024: SalioGen’s Nonviral In Vivo Gene Insertion Technology Takes Preclinical Strides
genengnews.com
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New Guidances Say Gene Therapies Require Strong Preclinical Testing . . Strong nonclinical testing should underpin development programs for both gene editing (GE) products and chimeric antigen receptor (CAR-T) cellular products, according to two new final guidances issued by the FDA. Since both of these therapeutics involve genetic alterations of living human cells and potentially, the introduction of viral vectors, sponsors need a firm foundation of in vitro and in vivo testing showing that they are safe and exert limited off-target activity, both guidances say. CAR-T for non-oncology indications is not covered. The guidance “Considerations for the Development of Chimeric Antigen Receptor (CAR) T Cell Products” focuses on the manufacturing and characteristics of CAR-T cells for oncology indications, including hematologic malignancies and solid tumors. Gene editing products are complex products that can include the gene editor, DNA targeting elements and a donor DNA template, according to “Human Gene Therapy Products Incorporating Human Genome Editing. #newguidance #genetherapy #preclinicaltesting #regulation #regulationupdate #regulatorycompliance
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Big news in vision health: Shannon Boye, PhD, receives FDA Clearance of IND Application to advance potential treatment for vision loss to help patients living with X-linked Retinoschisis (XLRS). Harrington Scholar Shannon Boye, PhD, University of Florida College of Medicine, serves as Founder and Director of Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness. XLRS is a rare disease that causes vision loss, and currently, there is no approved treatment. ATSN-201 utilizes a novel AAV capsid called AAV.SPR, which safely delivers the RS1 gene to the central retina, helping photoreceptors function properly. This gene therapy offers hope for those affected by XLRS, potentially reversing or preventing blindness. By overcoming challenges in previous treatments, ATSN-201 is a significant advancement in the field of genetic medicine. With the FDA's clearance, Atsena Therapeutics is set to start a Phase I/II clinical trial, called the Lighthouse Study, to evaluate ATSN-201's safety and efficacy in male patients with XLRS. This exciting progress brings us one step closer to transforming the lives of those affected by this debilitating disease. Congratulations to Dr. Shannon Boye and Atsena Therapeutics on this remarkable achievement! Harrington Discovery Institute is proud to be part of the journey, fostering groundbreaking research to improve vision and make a difference in patients' lives. Read More: https://bit.ly/3Kngwak #GeneTherapy #VisionHealth #MedicalInnovation #HarringtonScholar #XlinkedRetinoschisis #InnovativeResearch
Atsena Therapeutics Receives FDA Clearance of IND Application for ATSN-201, an Investigational Gene Therapy for the Treatment of X-linked Retinoschisis | May 01, 2023 | Harrington Discovery Institute at University Hospitals
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BioPharma & HealthTech Competitive Strategy & Insights | Digital & AI Solutions | Gene & Cell Therapy | Vaccines
Gene&Cell Therapy >> Edgewood's $20M Series A; Nanoscope to pursue FDA approval for eye gene therapy later this year: Plus, news about bluebird bio, Q32 Bio and Aquestive Therapeutics: Edgewood Oncology’s $20M Series A: The Brookline, MA-based biotech plans to use the money to support Phase 2a studies in acute myeloid leukemia and targeted breast cancer. It’s financed by Alta Partners. — Kyle LaHucik Nanoscope to seek FDA approval for eye gene therapy: The Dallas-based biotech said its mutation-agnostic gene therapy MCO-010 passed a Phase 2b study in retinitis pigmentosa, significantly improving vision measured on an eye chart at one year at both the high and low doses compared to a sham control. It expects to submit an application for MCO-010 to the FDA in the second half of this year. — Lei Lei Wu Bluebird bio projects 85 to 105 patient starts for its gene therapies in 2024: So far, the company has reported seven patient starts for beta thalassemia treatment Zynteglo and two for cerebral adrenoleukodystrophy therapy Skysona. Bluebird said that the first patient start for its sickle cell disease therapy is “imminent.” In its Tuesday earnings announcement, the company also shared that it signed an outcomes-based agreement with Michigan Medicaid, marking its first such agreement with a government payer. — Lei Lei Wu Q32 Bio begins trading: The mid-stage immune biotech completed its reverse merger with Homology Medicines and announced a $42 million private placement. Its shares $QTTB were down about 4% on Tuesday morning, its first day of trading on the Nasdaq. — Kyle LaHucik Aquestive Therapeutics closes $75M offering: The New Jersey biotech, which is developing an oral film to deliver medications, raised the money via a sale of 16.6 million shares at $4.50 apiece. — Kyle LaHucik #lucidquest #genetherapy #celltherapy
Edgewood's $20M Series A; Nanoscope to pursue FDA approval for eye gene therapy later this year
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Casgevy gets the green light for sickle cell disease and beta thalassemia using CRISPR/Cas9 technology. A groundbreaking one-time treatment, it's a game-changer for blood disorders. However, don't expect a flood of approvals soon – most CRISPR treatments are in early stages. According to GlobalData, only 12% are in Phase II, one in Phase III. Analysts suggest another CRISPR approval might not be on the horizon soon. Exciting times for gene therapy, but patience is key! #Casgevy #GeneTherapy #OBI4You https://zurl.co/PKWc
What’s Next for CRISPR?
biospace.com
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Senior Medical and Scientific Affairs Executive | Global Drug Development | Strategic Leader | Rare Disease Innovator | Transforming Patients' Lives
#Pfizer reported results from a phase 3 trial, #CIFFREO, evaluating an investigational recombinant adeno-associated virus #genetherapy known as fordadistrogene movaparvovec among ambulatory boys ages 4 to 7 years with #Duchenne muscular dystrophy. CIFFREO failed to hit its primary goal of improving #motorfunction when compared to placebo at one year of treatment, as assessed by a movement scale known as the #NorthStarAmbulatoryAssessment. Key secondary endpoints, such as 10-meter run/walk velocity and #timetorise, also failed to demonstrate a significant difference between fordadistrogene movaparvovec and placebo. These results come a little more than a month after Pfizer reported the death of a young boy participating in a related phase 2 trial called DAYLIGHT, which included boys with DMD ages 2 to 3 years. The boy had received the gene therapy in early 2023 and died from cardiac arrest on May 3 of this year. It will be interesting to review more detailed results from both of these studies to better understand the reasons why the trial failed and more importantly, whether the death of the young boy was related to the treatment. These learnings will be critical in improving future #clinicalresearch and #development of gene therapies for DMD. #DMD #AAVgenetherapy #musculardystrophy #adverseevent #drugdevelopment #challenges
UPDATE: Pfizer's phase 3 gene therapy trial fails to improve function for boys with Duchenne muscular dystrophy
fiercebiotech.com
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🧬 Regeneron Pharmaceuticals and Intellia Therapeutics are taking a significant step forward in medical research. They're broadening their collaboration to create in vivo CRISPR-based gene editing therapies, with a focus on tackling neurological and muscular diseases.The potential of this partnership could reshape the future of medical treatments. Interested in the breakthroughs of gene editing?👉 Click the link below for the full story! 📲 https://lnkd.in/eHeKE2-b #Regeneron #Intellia #CRISPR #GeneEditing #MedicalInnovation #NeurologicalDiseases #MuscularDiseases #MedicalResearch #TherapeuticAdvancements #FutureOfMedicine
Regeneron and Intellia expand collaboration on gene editing therapies - Westfair Communications
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Creativity is as important as knowledge / Director, Center for Genetics and Genomics / Director, Ph.D. Program in Sciences and Innovation in Medicine at Universidad del Desarrollo
What is the current state of cell and gene therapies? There are over 100 approved gene, cell, and RNA therapies worldwide, with an additional 3,700 in various stages of clinical and preclinical development. A recently published review in Molecular Therapy provides a comprehensive overview of the current landscape of advanced therapies. This includes viral and non-viral gene therapies, gene-editing cell therapies, mRNA therapeutics, antisense oligonucleotides, and reprogrammed cellular vaccines, among others. https://lnkd.in/eMD3p6A3 #genetics #genomics #precisionmedicine #genomicmedicine #geneediting #rna #vaccines #immunology #immunotherapy #immunity #cancer #raredisease #clinicaltrials #clinicalresearch #brain #eye #heart #liver #biotechnology #pharma #drugdevelopment #innovation #research #science #sciencecommunication
The state of cell and gene therapy in 2023
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What's next for CRISPR in 2024, and what are its main challenges coming into the New Year? Jasper Morley discusses below: “Challenges in this space include how to develop a fair pricing strategy, the logistics of offering broad access to the most needy populations in underserved markets (such as sickle cell patients in Africa), and potential unfamiliar adverse events associated with this novel modality of therapy. Vertex and CRISPR’s Casgevy will run $2.2 million for a one-time treatment for both SCD and TDT. That sticker price is in line with other novel gene therapies, such as bluebird bio’s Lyfgenia, which was also approved by the FDA for SCD and will cost $3.1 million. Yet one study of SCD patients found the total cost of disease over a patient’s lifetime was over $5 million." Read more below:
What’s Next for CRISPR? | BioSpace
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With 15 patients started across its three gene therapies, bluebird bio claims a nearly 140% year-over-year revenue growth and aims to initiate up to around 100 new patients in the current year. The Massachusetts-based biotech revealed that it has so far initiated 15 patients on its gene therapies, one of whom started the recently approved sickle cell disease treatment Lyfgenia just this month. The remaining 14 patients are receiving either Zynteglo, for pediatric beta-thalassemia, or Skysona, which is intended for cerebral adrenoleukodystrophy in boys. William Blair analyst Sami Corwin, Ph.D. anticipates that Lyfgenia will face stiff competition from CRISPR Therapeutics and Vertex Pharmaceuticals’ Casgevy, which the analyst predicts will be a “headwind” for bluebird. One key advantage for Casgevy is its price. Bluebird gave Lyfgenia a price tag of $3.1 million, nearly $1 million more than Casgevy’s $2.2 million. #biotech #biopharma #sicklecelldisease #genetherapy
Bluebird Boasts Nearly 140% Revenue Jump, Still Misses Target | BioSpace
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