# gene therapies (blogs) — RSS Amplifier Recent posts from the 14 feeds in the RSS Amplifier directory that cover gene therapies. Page: Feed: --- ## [Cleared 510(k) Submissions with Supporting Documents - 2026](http://www.fda.gov/vaccines-blood-biologics/substantially-equivalent-510k-device-information/cleared-510k-submissions-supporting-documents-2026) _2026-08-21 · FDA · Whats New Vaccines Blood Biologics RSS Feed_ Cleared 510(k) Submissions with Supporting Documents - 2026 ## [510(k) Blood Establishment Computer Software - 2026](http://www.fda.gov/vaccines-blood-biologics/substantially-equivalent-510k-device-information/510k-blood-establishment-computer-software-2026) _2026-08-21 · FDA · Whats New Vaccines Blood Biologics RSS Feed_ Listing of 510(k) Blood Establishment Computer Software for the year 2026 ## [BK251307- Donor Information System Version 9.0](http://www.fda.gov/vaccines-blood-biologics/substantially-equivalent-510k-device-information/bk251307-donor-information-system-version-90) _2026-08-21 · FDA · Whats New Vaccines Blood Biologics RSS Feed_ The Donor Information System (DIS) is used to manage donation center operations including but not limited to screening of donors for eligibility, immunizations, source plasma collection processing, and determining unit suitability. ## [Lawmakers urge FDA to enact new China policies after 3 gene therapy deaths](https://www.biospace.com/policy/lawmakers-urge-fda-to-enact-new-china-policies-after-3-gene-therapy-deaths) _2026-08-21 · Gabrielle Masson · FDA News: Approvals, Adcomms, Complete Response Letters and Guidances_ Three fatalities occurring recently in China clinical trials have spurred two members of U.S. Congress to urge the FDA to enact new policies designed to protect American patients. ## [The cost of waiting: what licensing a biotech asset early really saves you](https://www.labiotech.eu/opinion/cost-of-waiting-licensing-biotech-asset-early/) _2026-08-21 · Dylan Kissane · Labiotech.eu_ In recent years, biopharma has paid significantly different prices for the same underlying technology. In March 2025, AstraZeneca agreed to pay EsoBiotec, an in vivo CAR-T platform with early single-patient data, $425 million upfront and up to $575 million more in milestones. In August 2025, Gilead’s Kite paid $350 million for Interius BioTherapeutics, another in vivo CAR-T platform, also… ## [Biologics Procedures (SOPPs)](http://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics/biologics-procedures-sopps) _2026-08-21 · FDA · Whats New Vaccines Blood Biologics RSS Feed_ These Standard Operating Procedures and Policies (SOPPs) are used by staff in FDA's Center for Biologics Research and Review (CBER) in the performance of their duties. They are made available here to the public because CBER is committed to being as transparent as possible in how it operates. ## [Sickle cell pain crisis: A caregiver’s guide to home management](https://sicklecellanemianews.com/sickle-cell-crisis-home-management/) _2026-08-21 · Oluwatosin Adesoye · Sickle Cell Disease News – The Web's Daily Resource for Sickle Cell Disease News_ The first signs of a sickle cell disease pain crisis can spark instant panic. But not every pain crisis requires rushing to the emergency room. Some mild to moderate sickle cell pain episodes can be safely managed at home if the person is old enough to communicate their symptoms, their pain responds to prescribed home \[…\] The post Sickle cell pain crisis: A caregiver’s guide to home management… ## [Beyond biology: Nanobiotix’s physics-first approach to cancer](https://www.labiotech.eu/podcast/beyond-biology-nanobiotixs-physics-first-approach-to-cancer/) _2026-08-21 · Jules Adam · Labiotech.eu_ What if the key to beating cancer was a new way of thinking about matter? That's the question Laurent Lévy asked when he co-founded Nanobiotix over twenty years ago The post Beyond biology: Nanobiotix’s physics-first approach to cancer appeared first on Labiotech.eu . © Labiotech UG and Labiotech.eu. Unauthorized use and/or duplication of this material without express and written permission from… ## [Frequently Asked Questions — Developing Potential Cellular and Gene Therapy Products](http://www.fda.gov/regulatory-information/search-fda-guidance-documents/frequently-asked-questions-developing-potential-cellular-and-gene-therapy-products) _2026-08-20 · FDA · Whats New Vaccines Blood Biologics RSS Feed_ This is the guidance Frequently Asked Questions — Developing Potential Cellular and Gene Therapy Products ## [Potency Assessment of Active Immunotherapy Products](http://www.fda.gov/regulatory-information/search-fda-guidance-documents/potency-assessment-active-immunotherapy-products) _2026-08-20 · FDA · Whats New Vaccines Blood Biologics RSS Feed_ This is the draft guidance Potency Assessment of Active Immunotherapy Products ## [Trade Smarter With IBKR (Sponsored)](https://crawlproof.com/a/j2zn1A3oeRao) _2026-08-20 · **Sponsored**_ Join Interactive Brokers through a referral and access low-cost global trading. ## [Regeneron wins FDA approval to challenge Ipsen for rare bone disease market](https://www.biospace.com/fda/regeneron-wins-fda-approval-to-challenge-ipsen-for-rare-bone-disease-market) _2026-08-20 · Nick Paul Taylor · FDA News: Approvals, Adcomms, Complete Response Letters and Guidances_ Regeneron’s Pasatru is the second fibrodysplasia ossificans progressiva drug approved in the U.S., following the 2023 approval of Ipsen’s Sohonos. ## [Ultragenyx bags first gene therapy approval as previously rejected Sanfilippo asset awaits decision](https://www.biospace.com/fda/ultragenyx-bags-first-gene-therapy-approval-as-previously-rejected-sanfilippo-asset-awaits-decision) _2026-08-20 · Nick Paul Taylor · FDA News: Approvals, Adcomms, Complete Response Letters and Guidances_ The accelerated greenlight for Ultragenyx’s gene therapy for glycogen storage disease has raised analyst expectations for approval of UX111, which the FDA rejected last summer and is currently reviewing for a second time. A decision is expected by mid-September. ## [Why pediatric rare oncology outperforms in a volatile biotech market](https://www.biospace.com/drug-development/opinion-why-pediatric-rare-oncology-outperforms-in-a-volatile-biotech-market) _2026-08-20 · Paul Romness · FDA News: Approvals, Adcomms, Complete Response Letters and Guidances_ Real progress happens where unmet medical need is greatest and patient impact can be clearly measured. Pediatric rare cancers exemplify these criteria and have therefore generated continued investment, even in a capital-constrained environment. ## [eSubmitter Application History](http://www.fda.gov/industry/fda-esubmitter/esubmitter-application-history) _2026-08-19 · FDA · Whats New Vaccines Blood Biologics RSS Feed_ History of all updates to the application software in reverse chronological order by version number ## [FDA Approves First Therapy for Patients aged 8 years and older with Glycogen Storage Disease Type Ia](http://www.fda.gov/news-events/press-announcements/fda-approves-first-therapy-patients-aged-8-years-and-older-glycogen-storage-disease-type-ia) _2026-08-19 · FDA · Whats New Vaccines Blood Biologics RSS Feed_ The U.S. Food and Drug Administration today issued an accelerated approval for Genglycos (pariglasgene brecaparvovec-opnr) as the first-ever treatment for adults and children 8 years of age and older with glycogen storage disease type Ia (GSDIa) ## [Biologics Electronic Reading Room (eFOIA)](http://www.fda.gov/about-fda/center-biologics-evaluation-and-research-cber/biologics-electronic-reading-room-efoia) _2026-08-19 · FDA · Whats New Vaccines Blood Biologics RSS Feed_ The Biologics eFOI Reading Room provides access to documents posted throughout the "Vaccines, Blood & Biologics" and other sections related to the Center for Biologics Evaluation and Research on the FDA.gov Web site. ## [Trump's FDA pick Overton good for psychedelics, new pain meds but bad for vaccines: Analysts](https://www.biospace.com/fda/trumps-fda-pick-overton-good-for-psychedelics-new-pain-meds-but-bad-for-vaccines-analysts) _2026-08-19 · Heather McKenzie · FDA News: Approvals, Adcomms, Complete Response Letters and Guidances_ If confirmed by the Senate, Heidi Overton—officially revealed Wednesday as President Donald Trump’s choice for FDA commissioner—is expected to be a boon for developers of psychedelic therapeutics and non-opioid pain drugs, but could tow the party line on vaccines. ## [ASCENIV](http://www.fda.gov/vaccines-blood-biologics/approved-blood-products/asceniv) _2026-08-19 · FDA · Whats New Vaccines Blood Biologics RSS Feed_ Indicated for the treatment of Primary Immunodeficiency Diseases (PIDD) ## [Patient Listening Session Summaries](http://www.fda.gov/patients/fda-patient-listening-sessions/patient-listening-session-summaries) _2026-08-19 · FDA · Whats New Vaccines Blood Biologics RSS Feed_ Patient Listening Session summaries are published after each session to share a high-level summary of the discussion. The Public Engagement Staff draft summaries for FDA-requested sessions and the patient community requester drafts summaries for Patient-led sessions. ## [ELEVIDYS](http://www.fda.gov/vaccines-blood-biologics/tissue-tissue-products/elevidys) _2026-08-19 · FDA · Whats New Vaccines Blood Biologics RSS Feed_ Treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene. ## [Home Internet From Space (Sponsored)](https://crawlproof.com/a/A4CPCcVKzFqP) _2026-08-19 · **Sponsored**_ Residential satellite internet that brings connectivity to homes beyond conventional networks ## [Delayed puberty and other sickle cell challenges that aren’t widely discussed](https://sicklecellanemianews.com/columns/delayed-puberty-sickle-cell-challenges-widely-discussed/) _2026-08-19 · Oluwatosin Adesoye · Sickle Cell Disease News – The Web's Daily Resource for Sickle Cell Disease News_ Previously, I’d never publicly shared my experiences with delayed puberty and how deeply it affected me. Looking back, I realize that it was an important part of my journey with sickle cell disease (SCD). I hope my story will help young warriors and caregivers understand this often-overlooked complication and remind them that they are not \[…\] The post Delayed puberty and other sickle cell… ## [Life science industry in Geneva on a roll](https://www.labiotech.eu/in-depth/life-science-industry-in-geneva-on-a-roll/) _2026-08-19 · Roohi Mariam Peter · Labiotech.eu_ Geneva’s life science industry is bound to western Switzerland's Health Valley ecosystem but is also growing in and of itself. The post Life science industry in Geneva on a roll appeared first on Labiotech.eu . © Labiotech UG and Labiotech.eu. Unauthorized use and/or duplication of this material without express and written permission from this site’s author and/or owner is strictly prohibited.… ## [Trump picks White House aide Heidi Overton as FDA chief: Reports](https://www.biospace.com/fda/trump-picks-white-house-aide-heidi-overton-as-fda-chief-reports) _2026-08-19 · Annalee Armstrong · FDA News: Approvals, Adcomms, Complete Response Letters and Guidances_ If Heidi Overton clears the Senate nomination process, analysts say she will have a tough job ahead to “restore predictability” to an agency battered by layoffs and controversial policies. ## [Amylyx’s redemption story, Summit’s survival woes, Definium’s anxiety win, more](https://www.biospace.com/fda/amylyxs-redemption-story-summits-survival-woes-definiums-anxiety-win-more) _2026-08-19 · Jef Akst · FDA News: Approvals, Adcomms, Complete Response Letters and Guidances_ Amylyx shares good news for its investigational GLP-1 blocker; Definium announces positive data for its LSD-based therapy in anxiety; Summit/Akeso’s ivonescimab shows degrading survival benefits; patients treated with Neurocrine’s Prader-Willi drug experience serious adverse events; and Eli Lilly cracks down on the black market for next-gen obesity asset retatrutide. ## [Biologics PREA Reviews and Labeling Changes](http://www.fda.gov/about-fda/center-biologics-evaluation-and-research-cber/biologics-prea-reviews-and-labeling-changes) _2026-08-18 · FDA · Whats New Vaccines Blood Biologics RSS Feed_ PREA Labeling Changes, Medical, Statistical, and Clinical Pharmacology Reviews of Pediatric Studies Conducted under Section 505B of the Federal Food, Drug, and Cosmetic Act (the Act), as amended by the FDA Amendments Act of 2007 (FDAAA). These assessments are for CBER regulated drugs approved under ## [FDA Roundtable: Strengthening the Supply Chain for Blood Manufacturing - 09/29/2026](http://www.fda.gov/vaccines-blood-biologics/workshops-meetings-conferences-biologics/fda-roundtable-strengthening-supply-chain-blood-manufacturing-09292026) _2026-08-18 · FDA · Whats New Vaccines Blood Biologics RSS Feed_ On September 29, 2026, the Food and Drug Administration’s, Center for Biologics Evaluation and Research, Office of Blood Research and Review is hosting a roundtable on strengthening the supply chain for the collection and manufacturing of Whole Blood and blood components for transfusion and further manufacturing, including Source Plasma. ## [Approved Cellular and Gene Therapy Products](http://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products) _2026-08-18 · FDA · Whats New Vaccines Blood Biologics RSS Feed_ Listing of licensed and approved products from the Office of Therapeutic Products (OTP). ## [Review highlights non-opioid options for sickle cell pain management](https://sicklecellanemianews.com/news/review-highlights-non-opioids-sickle-cell-pain-management/) _2026-08-18 · Steve Bryson, PhD · Sickle Cell Disease News – The Web's Daily Resource for Sickle Cell Disease News_ Common pain relievers, such as nonsteroidal anti-inflammatory drugs (NSAIDs) or acetaminophen, combined with standard opioid care, may provide meaningful relief for acute pain in people with sickle cell disease (SCD), according to a review study. Cognitive behavioral therapy, a type of talk therapy, also showed signs of efficacy against chronic SCD pain, while other treatments, \[…\] The post Review… ## [EZPLAZ](http://www.fda.gov/vaccines-blood-biologics/blood-blood-products/ezplaz) _2026-08-18 · FDA · Whats New Vaccines Blood Biologics RSS Feed_ Freeze Dried Plasma ## [OTP Town Hall: Gene Therapy Chemistry, Manufacturing, and Controls – April 2023 - 04/25/2023](http://www.fda.gov/news-events/fda-meetings-conferences-and-workshops/otp-town-hall-gene-therapy-chemistry-manufacturing-and-controls-april-2023-04252023) _2026-08-18 · FDA · Whats New Vaccines Blood Biologics RSS Feed_ The FDA’s Center for Biologics Evaluation and Research (CBER) Office of Therapeutic Products (OTP) is hosting its next virtual town hall on Tuesday, April 25, 2023. ## [Fast, secure international transfers (Sponsored)](https://crawlproof.com/a/FSPqdo5vXxwG) _2026-08-18 · **Sponsored**_ Bank transfer, cash pickup, mobile wallet — low fees and real-time tracking ## [OTP Town Hall: Cell Therapy Chemistry, Manufacturing, and Controls – June 2023 - 06/08/2023](http://www.fda.gov/news-events/fda-meetings-conferences-and-workshops/otp-town-hall-cell-therapy-chemistry-manufacturing-and-controls-june-2023-06082023) _2026-08-18 · FDA · Whats New Vaccines Blood Biologics RSS Feed_ This event will focus on cell therapy chemistry, manufacturing, and controls (CMC), including tissue-engineered medical products regulated by OTP, and will be a continuation of the December 2022 virtual town hall. ## [BIO-Europe 2026: Expanding the scope at Europe’s leading biotech partnering event](https://www.labiotech.eu/partner/bio-europe-2026-partnering-event/) _2026-08-18 · Vyara Siteva · Labiotech.eu_ BIO-Europe 2026 heads to Cologne this November, expanding from three tracks to seven to reach the full biotech and pharma C-suite. The post BIO-Europe 2026: Expanding the scope at Europe’s leading biotech partnering event appeared first on Labiotech.eu . © Labiotech UG and Labiotech.eu. Unauthorized use and/or duplication of this material without express and written permission from this site’s… ## [Regulatory Harmonization and Convergence](http://www.fda.gov/vaccines-blood-biologics/international-activities/regulatory-harmonization-and-convergence) _2026-08-17 · FDA · Whats New Vaccines Blood Biologics RSS Feed_ The Agency engages in a range of explicit harmonization initiatives, a subset of which includes the participation of CBER. ## [Exporting CBER-Regulated Products](http://www.fda.gov/vaccines-blood-biologics/compliance-actions-biologics/exporting-cber-regulated-products) _2026-08-17 · FDA · Whats New Vaccines Blood Biologics RSS Feed_ The Division of Case Management (DCM) within CBER's Office of Compliance and Biologics Quality (OCBQ) directs and coordinates CBER's export programs, including review of requests for export of unapproved CBER-regulated products. ## [Tissue Establishment Registration](http://www.fda.gov/vaccines-blood-biologics/biologics-establishment-registration/tissue-establishment-registration) _2026-08-17 · FDA · Whats New Vaccines Blood Biologics RSS Feed_ Establishments that manufacture human cells, tissues, and cellular and tissue-based products (HCT/Ps) regulated solely under section 361 of the Public Health Service (PHS) Act are required to register with FDA pursuant to 21 CFR Part 1271. Also, provided information on when to register update regist ## [Blood Establishment Registration and Product Listing](http://www.fda.gov/vaccines-blood-biologics/biologics-establishment-registration/blood-establishment-registration-and-product-listing) _2026-08-17 · FDA · Whats New Vaccines Blood Biologics RSS Feed_ All owners or operators of establishments that manufacture blood products are required to register with the FDA, pursuant to section 510 of the Federal Food, Drug, and Cosmetic Act, unless they are exempt under 21 CFR 607.65 ## [Collaborations and Partnerships](http://www.fda.gov/vaccines-blood-biologics/science-research-biologics/collaborations-and-partnerships) _2026-08-17 · FDA · Whats New Vaccines Blood Biologics RSS Feed_ CBER Research Collaborations and Partnerships ## [Vaccine Adverse Event Reporting System (VAERS) Questions and Answers](http://www.fda.gov/vaccines-blood-biologics/vaccine-adverse-events/vaccine-adverse-event-reporting-system-vaers-questions-and-answers) _2026-08-17 · FDA · Whats New Vaccines Blood Biologics RSS Feed_ VAERS is a national vaccine safety surveillance program created as an outgrowth of the National Childhood Vaccine Injury Act of 1986 (NCVIA) and is administered by the Food and Drug Administration (FDA) and Centers for Disease Control and Prevention (CDC). ## [Vaccine Adverse Events](http://www.fda.gov/vaccines-blood-biologics/report-problem-center-biologics-evaluation-research/vaccine-adverse-events) _2026-08-17 · FDA · Whats New Vaccines Blood Biologics RSS Feed_ Information about the Vaccine Adverse Event Reporting System (VAERS), which is a national vaccine safety surveillance program co-sponsored by the Food and Drug Administration (FDA) and the Centers for Disease Control and Prevention (CDC) to detect possible signals of adverse events associated with v ## [CYFENDUS](http://www.fda.gov/vaccines-blood-biologics/vaccines/cyfendus) _2026-08-17 · FDA · Whats New Vaccines Blood Biologics RSS Feed_ For use as a post-exposure prophylactic vaccine following suspected or confirmed exposure to Bacillus anthracis in persons 18 through 65 years of age when administered in conjunction with recommended antibacterial regimen. ## [Learning to keep moving forward, even when I lack motivation](https://sicklecellanemianews.com/columns/learning-keep-moving-forward-lack-motivation/) _2026-08-17 · Dunstan Nicol-Wilson · Sickle Cell Disease News – The Web's Daily Resource for Sickle Cell Disease News_ Recently, I’ve been struggling with a departure from my normal routine. I’ve written about how exercise and good nutrition have helped me manage my sickle cell disease, but I’ve been traveling a lot and have found myself thrown off balance in ways I didn’t anticipate. At first, I blamed it on the time difference. I \[…\] The post Learning to keep moving forward, even when I lack motivation appeared… ## [Eight biotech companies in Melbourne you should know about](https://www.labiotech.eu/best-biotech/biotech-companies-melbourne/) _2026-08-17 · Roohi Mariam Peter · Labiotech.eu_ Discover 8 biotech companies in Melbourne, ranging from commercial to preclinical-stage companies, advancing promising technologies. The post Eight biotech companies in Melbourne you should know about appeared first on Labiotech.eu . © Labiotech UG and Labiotech.eu. Unauthorized use and/or duplication of this material without express and written permission from this site’s author and/or owner is… ## [Drugs@FDA Data Files](http://www.fda.gov/drugs/drug-approvals-and-databases/drugsfda-data-files) _2026-08-17 · FDA · Whats New Vaccines Blood Biologics RSS Feed_ Drugs@FDA Downloadable Data File in ZIP format, Data Definitions, and Entity Relationship Diagram (ERD) ## [Growing up with sickle cell disease: What my parents did right](https://sicklecellanemianews.com/growing-up-what-my-parents-did-right/) _2026-08-17 · Mary Shaniqua · Sickle Cell Disease News – The Web's Daily Resource for Sickle Cell Disease News_ So, your child has been diagnosed with sickle cell disease. What now? Grieve, but don’t let it paralyze you. The greatest risk isn’t the grief itself, but how it can freeze parents in fear, denial, or helplessness. Because raising a child with sickle cell, however unfair, means you need to step up as a parent who \[…\] The post Growing up with sickle cell disease: What my parents did right appeared… ## [Coping with sickle cell caregiver burnout: Resources and support](https://sicklecellanemianews.com/sickle-cell-caregiver-burnout/) _2026-08-17 · Oluwatosin Adesoye · Sickle Cell Disease News – The Web's Daily Resource for Sickle Cell Disease News_ Note: Oluwatosin Adesoye is a practicing physician living with sickle cell disease and is a columnist for Sickle Cell Disease News. Caring for someone with sickle cell disease is an act of love that often happens quietly in emergency rooms and hospital wards, and during sleepless nights at home. Caregivers become advocates, medication reminders, chauffeurs, \[…\] The post Coping with sickle cell… ## [Moving from pediatric to adult sickle cell care: How to navigate the transition](https://sicklecellanemianews.com/pediatric-to-adult-sickle-cell-transition/) _2026-08-17 · Mary Shaniqua · Sickle Cell Disease News – The Web's Daily Resource for Sickle Cell Disease News_ Note: Mary Shaniqua lives with sickle cell disease and is a columnist for Sickle Cell Disease News. Growing up, I had excellent pediatric hematology care. It was consistent, I had a trusted specialist nurse, frequent appointments and testing, and so on. Everything changed when I transitioned to the adult clinic. Before I could even adjust, \[…\] The post Moving from pediatric to adult sickle cell… ## [How caregivers can advocate for their loved one at a sickle cell appointment](https://sicklecellanemianews.com/sickle-cell-appointment-advocacy/) _2026-08-17 · Dunstan Nicol-Wilson · Sickle Cell Disease News – The Web's Daily Resource for Sickle Cell Disease News_ Note: Dunstan Nicol-Wilson lives with sickle cell disease and is a columnist for Sickle Cell Disease News. A hematology appointment may last only 20 or 30 minutes, but the decisions made during that time can shape the months that follow. As a caregiver, your preparation and observations can help ensure those decisions reflect everyday life. \[…\] The post How caregivers can advocate for their loved… ## [August 2026](https://lexiventures.substack.com/p/august-2026) _2026-08-17 · Lexi Ventures · Lexi Ventures Newsletters_ AI at Lexi, An engineered virus, Turning a gene down, Experiments by robots, A living pesticide, Where the exits are ## [Protected: To Swim Among the Stars](https://logancollinsblog.com/2026/08/16/to-swim-among-the-stars/) _2026-08-17 · logancollins · LoganThrasherCollins.com_ There is no excerpt because this is a protected post. ## [Sudden Death Without Explanation in a Healthy 20-Year Old](https://robertchandler.substack.com/p/foreign-non-local-material-deposits) _2026-08-16 · Robert W. Chandler, MD MBA · Experimental Gene Therapy_ Case 152: SADS 1 = Found Dead, COF 4 = Unrelated Tissue Changes, COD 0 = Unrelated to Cause of Death, BNT162b2 180 and 141 days prior to death.