XTalks

SEPTEMBER 14, 2022

In this episode, Ayesha discussed the FDA approval of Sanofi’s enzyme replacement therapy Xenpozyme for the treatment of non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD), a rare genetic lysosomal storage disease, in adults and pediatric patients.

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XTalks

DECEMBER 22, 2021

If the past year is anything to go by, then 2022 will also be a year marked by continued innovations in the life sciences. The development and widespread adoption of new technologies is key to revolutionizing the way we diagnose, prevent, treat and manage disease. The RNA Revolution: From mRNA Vaccines to RNA Editing.

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Life Science RNA Vaccination Vaccine 96

STAT News

SEPTEMBER 12, 2022

In the life sciences industry, where company names derived from “biology” or “genetics” are a dime a dozen, it helps to have a brand that stands out. The company is developing treatments for diseases caused by broken mitochondria — the main energy source for human cells.

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Genetic Disease Life Science Genetics Branding 98

Pharmaceutical Technology

MAY 24, 2023

The AMP programme is a public-private partnership between a number of biopharmaceutical and life sciences companies, the US Food and Drug Administration (FDA) and the National Institutes of Health (NIH), along with non-profit and other organisations.

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Scienmag

JUNE 7, 2021

Credit: Autobahn Labs Cold Spring Harbor Laboratory (CSHL) is collaborating with Autobahn Labs, a new life sciences incubator, to catalyze the Laboratory’s early-stage discovery programs into spin-out companies that commercialize transformational new therapies. Autobahn will provide […].

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Genetic Disease Life Science Genetics Research 52

Pharmaceutical Technology

JANUARY 12, 2023

Under the research partnership deal, the companies will develop new precision genetic medicines for a severe form of genetic dilated cardiomyopathy (DCM). Our pipeline of programmes focused on neuromuscular and cardiac rare disease gives us deep insight into the diverse and complex nature of dilated cardiomyopathy.”.

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RNA Gene Therapy Genetics Genetic Disease 130

XTalks

JANUARY 27, 2023

The website brings awareness for facial angiofibroma, an aspect of the rare genetic disease tuberous sclerosis complex (TSC). Nobelpharma America, a pharmaceutical and medical device company headquartered in Bethesda, Maryland, has launched an educational website called Face Forward.

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Life Science Genetic Disease FDA Approval Marketing 97

World of DTC Marketing

JANUARY 24, 2022

A group of early-stage life science investors and biotech CEOs joined together with a prominent patient advocate to speak out against H.R.3, The panel was hosted by the life sciences venture capital coalition Incubate and held at the National Press Club. Only about $2.2 3, known as the Lower Drug Costs Now Act.

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XTalks

NOVEMBER 10, 2020

Expeditious and accurate diagnoses are necessary for patients to access healthcare services and treatment options for rare genetic diseases. Increasing the efficiency of case analysis and interpretation is essential to providing timely care for patients with genetic diseases.

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Genomics Genome Medicine Genetic Disease 98

XTalks

MAY 17, 2023

Related: Gene Therapy Clinical Trials for Rare Diseases: 5 Key Takeaways from FDA’s Recent Town Hall “The BGTC offers promise for rare disease patients around the world,” said Julie Gerberding, MD, CEO of the FNIH, in the press release from the agency. In the US, more than 30 million people live with a rare disease.

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XTalks

NOVEMBER 4, 2024

On Familial Chylomicronemia Syndrome (FCS) Awareness Day, which falls on November 1 every year, Arrowhead Pharmaceuticals launched a new campaign called ‘We’ll Get There Soon’ to shed light on the rare disease. The initiative aims to raise awareness about the disease and the challenges faced by patients living with the rare genetic disorder.

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XTalks

SEPTEMBER 13, 2021

With RNA therapies being the next hot thing in genetic medicine, Eli Lilly is joining the RNA editing race by partnering with Netherlands-based ProQR Therapeutics NV (Nasdaq: PRQR), a biotech company developing RNA-based therapies for rare genetic diseases with a focus on blinding disorders of the retina.

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RNA Genetics Genetic Disease Medicine 102

Pharmaceutical Technology

MAY 8, 2023

Innovation S-curve for the pharmaceutical industry Zinc-finger nucleases is a key innovation area in pharmaceutical Zinc finger nucleases (ZFNs) are tools used in genetic engineering to cleave a chosen genomic sequence. ZFNs are also used to create a new generation of genetic disease models called isogenic human disease models.

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Genetics DNA Genetic Engineering Antibody 130

XTalks

NOVEMBER 29, 2024

Govorestat is a central nervous system (CNS)-penetrant aldose reductase inhibitor designed to target a critical problem in classic galactosemia — a rare genetic condition that prevents proper metabolism of the sugar galactose.

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Drugs Compliance Clinical Trials Clinical Trials 79

XTalks

MAY 26, 2023

Vyjuvek is also the first drug approved to treat the disease and is Krystal’s first approved product. DEB is a genetic disorder characterized by very fragile skin that rips and blisters easily even from minor friction (like rubbing or scratching) or injury, resulting in open wounds that are prone to skin infections and fibrosis.

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Gene Therapy Gene FDA Approval Dermatology 89

XTalks

OCTOBER 16, 2024

This is Pfizer’s second FDA-approved treatment for a rare genetic blood disorder this year. Hympavzi’s approval, with its innovative approach to balancing clotting, could offer a modern solution that adds to the global effort to reduce the risk of thrombosis.

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XTalks

FEBRUARY 14, 2022

The UK’s National Health Service (NHS) has recommended the use of Libmeldy for the treatment of the rare genetic disease metachromatic leukodystrophy (MLD). He added that, “A deep body of evidence now points to the potential for durable effects in HSC gene therapy for certain severe genetic diseases including MLD.

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XTalks

DECEMBER 13, 2023

3) Fulgent Genetics Compound annual growth rate: 260 percent Fulgent Genetics, headquartered in California, is a technology-driven company with a strong presence in clinical diagnostics and therapeutic development. The company achieved core revenues of $67 million, reflecting a remarkable 48 perecent year-over-year growth.

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Genetics Vaccination Vaccine DNA 107

XTalks

FEBRUARY 28, 2022

In the US, 25 to 30 million Americans, or one in ten, are living with a rare disease. Are Most Rare Diseases Genetic? While 72 percent of rare diseases are genetic, not all are. Rare genetic diseases can be caused by either somatic or germline genetic changes.

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Clinical Trials Clinical Trials Genetics Trials 91

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells through epigenetic editing without altering the genetic sequence itself.

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DNA Gene Gene Silencing Genomics 97

XTalks

MAY 12, 2023

Rett syndrome is a rare genetic disorder that predominantly affects girls and leads to severe physical and cognitive impairments. Dr. Bishop: Rett syndrome is a genetic disorder caused by a mutation in the MECP2 gene. Rett syndrome is a neurodevelopmental disorder — not a neurodegenerative disease.

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FDA Approval Genetics Trials Drugs 89

XTalks

AUGUST 29, 2022

Using this novel technique, the team also demonstrated that they could generate over a billion CAR-T cells , which are T-cells that have been genetically modified to fight the cancer cells that cause multiple myeloma. Scientists have previously shown that they could replace small sections of the IL2RA gene where mutations occur in patients.

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Gene Editing DNA Gene Genomics 95

XTalks

FEBRUARY 27, 2023

Chiesi Global Rare Diseases recently announced in a press release that Lamzede (velmanase alfa-tycv) received US Food and Drug Administration (FDA) approval for the treatment of non-neurological manifestations of alpha-mannosidosis in both adult and pediatric patients.

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FDA Approval Life Science Containment Genetics 52

pharmaphorum

OCTOBER 16, 2020

Findacure is a charity that works directly with rare disease patient groups to help them grow and professionalise. Over the last five years we have focused on the power of drug repurposing for rare genetic diseases. The post Rare diseases, repurposing and the role of AI appeared first on.

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XTalks

SEPTEMBER 16, 2024

HAE is a rare genetic condition that causes episodes of severe swelling in various parts of the body, including the face, hands, feet and airway. These attacks can be unpredictable, often leading to life-threatening situations when they affect the throat or lungs.

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Protein Genetics Trials Allergies 52

XTalks

NOVEMBER 27, 2023

Pompe disease, also referred to as acid-maltase disease and glycogen storage disease II, is an uncommon genetic disorder characterized by the gradual weakening of both cardiac and skeletal muscles. Crowley, executive chairman of Amicus Therapeutics, in the company’s press release.

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FDA Approval Clinical Trials Clinical Trials Trials 96

XTalks

SEPTEMBER 12, 2022

ASMD is a rare genetic lysosomal storage disease caused by mutations in the sphingomyelin phophodiesterase-1 ( SMPD1 ) gene that codes for the acid sphingomyelinase (ASM) enzyme. ASM is required for breaking down a complex lipid called sphingomyelin.

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FDA Approval Genetics Trials Gene 97

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells without editing the genetic sequence itself. Related: 2020 Year in Review: COVID-19, CRISPR and Immunotherapies Define the Year for the Life Sciences.

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DNA Gene Gene Silencing Genomics 52

XTalks

SEPTEMBER 24, 2024

CDKL5 deficiency disorder (CDD) is a rare genetic condition caused by mutations in the CDKL5 gene, which is essential for brain development and function. This disorder leads to severe neurodevelopmental impairment and early-onset, difficult-to-control seizures.

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Clinical Trials Clinical Trials Trials Development 52

XTalks

APRIL 24, 2023

Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday.

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Gene Therapy Gene Trials Clinical Trials 81

XTalks

OCTOBER 7, 2020

A tiny child with a devastating genetic disease who wasn’t supposed to blow out the candles on his first birthday cake. “They called him ‘The Boy.’ Who wasn’t supposed to sit up. Not only did this baby survive to do all these things, but he became a poster child for gene therapy with the regulators at the U.S.

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Development Gene Therapy Clinical Trials Clinical Trials 98

XTalks

SEPTEMBER 22, 2022

GPP occurs in individuals with specific gene variations as it is a genetic disease , and variations in the genes IL36RN and AP1S3 are known to cause it. The novel, selective monoclonal antibody spesolimab behind Spevigo reduces flares in individuals by inhibiting interleukin-36 (IL-36) signalling.

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FDA Approval Clinical Trials Clinical Trials Dermatology 52

XTalks

SEPTEMBER 27, 2024

In a news release published yesterday, Pfizer announced it will voluntarily withdraw its sickle cell disease (SCD) therapy Oxbryta (voxelotor) from all markets worldwide. The company said it is also discontinuing all active clinical trials for voxelotor and expanded access programs around the world.

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Marketing Gene Therapy Sales Drugs 102

XTalks

APRIL 15, 2021

A 2015 study published in Nature Genetics found that the availability of human genetic data made investigational drugs twice as likely to pass pivotal trials and eventually be approved. Figure 1: The use of Mendelian randomization to validate genetic drug targets.

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Development Drugs Genomics Genome 95

XTalks

AUGUST 16, 2021

Immune systems can be weakened in individuals with HIV/AIDS; cancer and transplant patients taking certain immunosuppressive drugs; and those with genetic diseases affecting the immune system like congenital agammaglobulinemia and congenital IgA deficiency. According to the CDC, approximately 2.7

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Vaccination Vaccine Antibody Immune Response 94

Delveinsight

FEBRUARY 11, 2021

Verily, Google’s life-science-focused sibling company and Janssen will also seek to tap into the data generated by people during their everyday lives to seek for any previous health-related signals in the two years leading up to the point they consented to participate in the study as well as in the two years after.

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Gene Therapy Gene Immune Response In-Vivo 52

XTalks

MARCH 3, 2021

The US Food and Drug Administration (FDA) has granted approval to Nulibry (fosdenopterin) for reducing the risk of death associated with molybdenum cofactor deficiency type A (MoCD Type A), making it the first treatment for the ultra-rare, genetic metabolic disorder. The approval was granted to BridgeBio Pharma, Inc.

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