daniel-gordon/biopharma-dive huggingface dataset:
Neumora, a brain drug developer has advanced an experimental treatment for depression into latestage testing, and has a second drug for Alzheimers agitation in a Phase 1 trial. It was backed by private raising more than 600 million
Viking Sofinnova and Wellington Investments coled a 160 million Series D round for RayzeBio in September
We are committed to working collaboratively with the FDA and because the agency did not suggest that additional Phase 3 studies may be needed to demonstrate the products safety and efficacy we believe we are well prepared to address the ir concerns  said Jan Mikkelsen Ascendis Pharmas President and CEO
About Duchenne Muscular Dystrophy Duchenne muscular dystrophy DMD is a rare fatal neuromuscular genetic disease that occurs in approximately one in every 35005000 males worldwide
The startup is built on technology for profiling circulating chromatin and the DNA methylome in plasma samples
The company still intends to keep testing exarafenib in combination with other drugs like binimetinib a therapy sold by Pfizer under the brand name Mektovi
AEs reported in greater than or equal to 5 percent of patisiran patients and seen at least 3 percent more frequently with patisiran compared with placebo were infusionrelated reactions 122 percent vs 9 percent respectively arthralgia 77 percent vs 45 percent respectively and muscle spasms 66 percent vs 22 percent respectively
To date Moderna only has approval to sell its COVID19 vaccine
All statements other than statements of historical facts included in this press releas e regarding Ascendis future operations plans and objectives of management are forward looking statements
5 patients 28 percent on patisiran and 8 patients 45 percent on placebo died
Apellis to lay off 25 of staff trim research in major restructuring The workforce reduction and related spending cuts are meant to focus resources in support of the companyʼs new geographic atrophy drug Syfovre
Along the way the company has also been working toward its longterm vision of becoming a fullfledged biotech. The rest of the industry still hasn't fully caught on according to Anne Wojcicki the companys cofounder and CEO
Trivenis lead drug candidate by comparison targets kallikreins 5 and 7  enzymes implicated in skin barrier problems
Abrahams and his team therefore estimate that FDA approval for a subcutaneous version would add more than 1 billion to Leqembis peak annual sales which RBC has pegged at 10 billion
ATTR amyloidosis with cardiomyopathy is an increasingly recognized cause of heart failure aecting greater than 250000 patients around the world
Verve has been running that study in the UK and New Zealand and next month will present initial data at a medical conference being held in Philadelphia
Under the new agreement, Orionis will discover and optimize the experimental medicines for targets selected by Genentech
Sarepta does not undertake any obligation to publicly update its forwardlooking statements based on events or circumstances after the date hereof except as required by law
The company had cash and cash equivalents of about 616 million at the end of the second quarter enough to fund operations through early 2025
Taysha Gene Therapies Inc Condensed Consolidated Balance Sheet Data in thousands except share and per share data Unaudited June 30 2023December 31 2022 ASSETS Current assets Cash and cash equivalents  45083 87880 Prepaid expenses and other current liabilities 9032 8537 Total current assets 54115 96417 Restricted cash 2637 2637 Property plant and equipment net 14139 14963 Operating lease rightofuse assets 10348 10943 Other noncurrent assets 304 1316 Total assets  81543 126276 LIABILITIES AND STOCKHOLDERS DEFICIT EQUITY Current liabilities Accounts payable  10766 10946 Accrued expenses and other current liabilities 19631 18287 Deferred revenue 26909 33557 Total current liabilities 50641 62790 Deferred revenue net of current portion 6212 Term loan net 38354 37967 Operating lease liability net of current portion 19528 20440 Other noncurrent liabilities 3922 4130 Total liabilities 118657 125327 Stockholders deficit equity Preferred stock 000001 par value per share 10000000 shares authorized and no shares issued and outstanding as of June 30 2023 and December 31 2022   Common stock 000001 par value per share 200000000 shares authorized and 64432637 and 63207507 issued and outstanding as of June 30 2023  and December 31 2022  respectively 1 1 Additional paidin capital 406546 402389 Accumulated deficit 443661 401441 Total stockholders deficit equity 37114 949 Total liabilities and stockholders deficit equity  81543 126276 Taysha Gene Therapies Inc Condensed Consolidated Statements of Operations in thousands except share and per share data Unaudited For the three months ended June 30 2023For the three months ended June 30 2022For the six months ended June 30 2023For the six months ended June 30 2022 Revenue Service Revenue  2395    7101   Operating expenses Research and development 19791 23506 32305 61688 General and administrative 5988 9867 14739 21336 Total operating expenses 25779 33373 47044 83024 Loss from operations 23384 33373 39943 83024 Other income expense Interest Income 223 27 542 41 Interest expense 1440 743 2814 1415 Other expense 3 3 5 11 Total other income expense 1214 719 2277 1385 Net loss  24598 34092 42220 84409 Net loss per common share basic and diluted  038 085 066 216 Weighted average common shares outstanding basic and diluted 64244531 40142403 63755435 39163996 Company Contact Hayleigh Collins Director Head of Corporate Communications Taysha Gene Therapies Inc hcollinstayshagtxcom Media Contact Carolyn Hawley Canale Communications carolynhawleycanalecommcom Source Taysha Gene Therapies Inc
While Beam will continue to conduct discovery research Evans added that the company could not advance every idea on its own
idsmartlinkurlhttps3A2F2Fwwwalnylamcom2Fesheet52800209newsitemid2 0220803005528lanen USanchorAlnylamPharmaceuticals2CIncindex1md565b7fd906d15b70baf323ab6f b86f4afNasdaq ALNY the leading RNAi therapeutics company today announced that the APOLLOB Phase 3 study of patisiran an investigational RNAi therapeutic in development for the treatment of transthyretinmediated ATTR amyloidosis with cardiomyopathy met the primary endpoint of change from baseline in the 6Minute Walk Test 6MWT at 12 months compared to placebo pvalue 00162
Biogen decided to invest in the newer medicine then known as SAGE217 or zuranolone back in 2020 at the same time it acquired rights to SAGE324 seen as a treatment for essential tremor and other neurological disorders
The study also met the first secondary endpoint of change from baseline in quality of life compared to placebo as measured by the Kansas City Cardiomyopathy Questionnaire KCCQ pvalue 00397
Sanofi invests in MeiraGTx and its generegulating technology. The pharma is putting 30 million into the London and New Yorkbased biotech which signaled other deals could be coming soon
Important Safety Information What is VOXZOGO used for
At a median of week 12 30 mgkg of SRP5051 resulted in mean dystrophin production of 655 of normal
In the primary functional endpoint SRP9001treated participants showed an increase in NSAA total score compared to placebo at 48 weeks however the difference was not statistically significant P037
Among the deals top prospects is a Phase 3 vaccine candidate for the mosquitoborne chikungunya virus that showed promise in data published last month
The companies that IPO successfully have a profile RayzeBio and Neumora share strong private investor backing veteran leadership and at least one drug candidate in or close to clinical trials
They also determined the companys manufacturing data were grossly deficient and cited missing biomarker results in dismissing afterthefact analyses that BrainStorm conducted to support its application. Those concerns led the FDA in November to turn back BrainStorms application issuing the company a refuse to file letter
But we also have a very large therapeutic side thats focused on how were going to translate this data to the benefit of our customers
Metachromatic leukodystrophy affects about 1 in every 100000 newborns or fewer than 50 new cases per year in the US Its caused by a mutation that results in an accumulation of fat in the brain and other organs in turn causing neurological problems and death. It is one in a family of diseases called  lysosomal storage disorders  that have been targeted by gene therapy developers
The condition is universally fatal and death usually occurs before the age of 30 due to respiratory or cardiac failure
Bluerock a Massachusetts-based subsidiary of the German pharmaceutical giant Bayer said its stem cell-derived therapy met the studys main goal appearing safe and well-tolerated in the 12 patients who received it
Reporter: Do you still feel your company is an outsider in the healthcare system? CEO:  I feel like we are getting closer. For a while we were on the outside and some of the worst things our competitors did was coin the term recreational genomics which was just awful as though all we do are cilantro studies and Neanderthal reports. But we were also so dangerous that we had to be shut down and regulated
Over a fouryear period children aged 2 years and above who received VOXZOGO exhibited a mean average height Zscore improvement of 11 to 14 standard deviations 95 CI limits from 046 to 193 and a mean height gain of 63 to 78 centimeters cm 95 CI limits from 298 to 1040 cm when compared to untreated children with achondroplasia of the same age and sex
It envisions the therapy as a longlasting preventive therapy delivered through a hemophilia A Vega expects to complete the study by the end of 2024  according to a federal clinical trials database
We look forward to having continued discussions with the FDA with the goal of bringing TSHA102 to patients as safely and expeditiously as possible TSHA102 is being evaluated in the REVEAL Phase 12 adult trial in Canada
Sarepta does not undertake any obligation to publicly update its forwardlooking statements based on events or circumstances after the date hereof
The decrease in selling general and administrative expenses primarily reflects the following 162 million decrease in professional service expenses primarily due to a decrease in reliance on thirdparty selling general and administrative contractors as well as a transaction fee for the Roche transaction incurred during the six months ended June 30 2020 with no similar activity incurred during the six months ended June 30 2021 and 22 million increase in stockbased compensation primarily due to changes in stock price
Third quarter revenue reached 43 million, double the consensus estimates on Wall Street
Protein degradation is a relatively new research area in biotech with companies like Nurix Kymera Therapeutics and C4 Therapeutics going public in 2020 and others like Seed Therapeutics and Lycia Therapeutics winning partnerships with Eli Lilly
FDAs decision to hold a public advisory committee meeting on the SRP 9001 BLA is a change from the communicated position at the midcycle meeting
The multi-billiondollar investment is a positive sign for a biotech sector still looking to shake off a down market
The new Novo foundationbacked cell therapy plant will serve more than the drugmakers projects however, tt aims to be able to aid in the manufacturing of multiple types of cells including embryonic stem cells induced pluripotent stem cells and adult optimization of manufacturing processes quality assurance and regulatory support
The 50 mgkg once weekly dosing regimen of EXONDYS 51 is not recommended
Amylyx was helped by a positive clinical trial result for its drug while the FDA and its advisers agreed Biogens drug showed an effect on levels of an important protein that are reasonably likely to predict a clinical benefit
Some patients were in confused states days after beginning treatment while others experienced unusual physical weakness or lack of energy
Should the FDA agree to a review the new version could be cleared and on the market by early 2025
This correlates to 4x increase in exon skipping compared to the 20 mgkg cohort of SRP5051 at 12 weeks mean exon skipping of 257 n2 and an 18x increase in exon skipping compared to a weekly 30 mgkg dose of eteplirsen at 24 weeks mean exon skipping of 059 n16
Ive talked to all kinds of people over the last year who stare at me thinking  you do research she said. She assures them that yes 23andMe does real drug research
We have already enrolled and dosed 11 participants in Study 103 using our commercial process material and we will have biomarker and safety results from that cohort in the second quarter
Fulcrum raised about 140 million in private funding from Third Rock Ventures and others and followed with a 72 million initial public offering in 2019
In the last two years a number of startups have emerged hoping to harness the CRISPR technology. Chroma Medicine is among the most wellfunded of the group raising 135 million in a Series B round earlier this year and just recently striking a development deal with Sangamo Therapeutics
Panelists also said companies need to be more disciplined with their financing and learn to be nimble when market down swings occur. Still many are hopeful the next year could see a different market for biotech
Conference Call The Company will be hosting a conference call at 430 pm Eastern Time to discuss Sareptas financial results and provide a corporate update
DMD is caused by a change or mutation in the gene that encodes instructions for dystrophin
After 16 weeks the group on izokibep didnt show a significantly better response than those getting a placebo Acelyrin said Monday
Intarcias medicine showed promise in clinical testing but the company hit roadblocks on its way to a potential FDA approval
A pivotal moment on that path was an FDA advisory committee meeting last November  when a panel of experts backed approval of the drug for kidney disease patients
A proofofconcept trial is expected to begin next year. The next step would be packaging the therapy in a device called SeraGraft to allow use in patients without the need for immunosuppressive medicines. Seraxis calls this combination SR 01
The passcode for the call is 8374717
Alfasigma executives said the transaction will help it develop its US presence. Intercept represents a compelling fit with hepatology and we believe that the transaction represents a transformational opportunity for both companies said Alfasigma CEO Francesco Balestrieri in a statement
The money will also enable continued progress on the companys other pipeline programs which include preclinical drugs targeting depression Parkinsons disease hyperactivity and impulsivity
Targeted radiation drugs such as Novartis drugs Pluvicto and Lutathera are more attractive than typical radiation therapy because the medicines can target cancer cells while sparing surrounding healthy cells
A biochemist by schooling, Stilz has been with Novo for more than a decade initially to oversee the drugmakers strategic partnerships. Previously he spent 17 years with Sanofi including a stint working to discover new diabetes drugs
On Oct 2 Jane Grogan will officially take over the position which has been without a permanent appointee for nearly two years. Grogan recently served as chief scientific officer at Graphite Bio a cell and gene therapy developer and as a scientific advisory board member for Bicycle Therapeutics a synthetic biology company. She also spent more than 15 years as a scientist at Genentech where she led research programs directed at cancer and disorders tied to the immune system. That experience proved attractive to Biogen leaders who have said they want to diversify research efforts that for much of the companys history have focused on diseases of the brain and central nervous system
Insulin light orange binding induces structural changes within the receptor which activates a signal cascade leading to the transport of glucose yellow into the cell through glucose transporter proteins red
Ascendis is headquartered in Copenhagen Denmark and has additional facilities in Heidelberg and Berlin Germany Palo Alto and Redwood City California and Princeton New Jersey
To qualify for the experiment patients have to have experienced at least four vaso occlusive crises  the painful episodes associated with the disease  within a year of screening or at least two within the last six months. Study participants must also have previously received one or possibly two other sickle cell medicines. The more targeted focus could mean a far smaller market opportunity for Fulcrum which is already facing a large group of 10000 patients in the US fit the new study criteria a fraction of the roughly 100000 in the country believed to have the disease
Forwardlooking statements include statements concerning the potential of our product candidates including the reproducibility and durability of any favorable results initially seen in our first patient dosed in the REVEAL trial and including our preclinical product candidates to positively impact quality of life and alter the course of disease in the patients we seek to treat our research development and regulatory plans for our product candidates the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies and whether if approved these product candidates will be successfully distributed and marketed the potential market opportunity for these product candidates our corporate growth plans statements associated with the timing size and completion of the Private Placement the forecast of our cash runway and the Companys expectations regarding funding operating and working capital expenditures
Biotech research labs are always churning out new drugs. But recently a few types of treatments have drawn more attention from investors.
Priovant is developing its drug brepocitinib in dermatomyositis and lupus
The Company uses these nonGAAP measures as key performance measures for the purpose of evaluating operational performance and cash requirements internally
The functional status at baseline for participants in the 45 age group was balanced across the placebo and treatment cohorts
Around 13 of DMD patients have mutations which make them amenable to skipping exon 51
Once identified Genentech will then take over on development and commercialization. PeptiDream retains its right to develop and commercialize its drug conjugates in Japan
ONPATTRO is an intravenously administered RNAi therapeutic targeting transthyretin TTR
Harbinger Health raises $140M to study bloodbased cancer screening test The test developer will use the Series B funding to complete a 10000 subject clinical trial ahead of a planned launch in 2025
The restructuring is a notable step back from Beam which until recently commanded a market value of several billion dollars on the promise of technology that could precisely change single DNA letters in the human genome
About SRP9001 delandistrogene moxeparvovec SRP9001 delandistrogene moxeparvovec is an investigational gene transfer therapy intended to deliver SRP9001 to muscle tissue for the targeted production of functional components of dystrophin
RayzeBio  which is developing drugs to more precisely deliver radiation into the body filed to go public one day before Neumora
Rapport said all of its existing investors participated too including Third Rock Arch and Johnson Johnson Innovation
Todays advisory committee outcome is extremely important to the patient community who are in urgent need of new therapies said Doug Ingram  president and chief executive officer Sarepta
Clinical testing has found exacel alleviates the most serious and impactful effects of sickle cell as well as another rare blood disorder betathalassemia
At least 11 developers of gene editing or gene replacement therapies have laid off staff in 2023 including Beam Therapeutics  from which Verve licenses its technology
The company also signaled more transactions may be coming in the future after being approached by multiple other companies interested in its work
The Novo Nordisk Foundation the controlling owner of the Danish drugmaker will spend up to 950 million kroner or roughly 136 million to build a cell therapy manufacturing facility that can support development of treatments for use in human testing according to an announcement Thursday
All but three of the biotechs that have gone public this year now trade below their offering price most notably Acelyrin which revealed a disappointing trial setback earlier this month
April 2023 Ascendis Pharma AS
Jj-backed startup raises another 150M for brain drug development. The funding for Rapport Therapeutics saw participation from a series of socalled crossover investors many of which have pulled back from biotech startups in recent years
Other conditions that could see benefit from cell therapies include chronic heart failure Type 1 diabetes Parkinsons disease and kidney disease according to the Novo foundation
The FDA turned back a second application earlier this year as Ocalivas only modest benefit was balanced against signs of liver damage experienced by some trial participants given the drug
The drug dubbed VTX958 actually met the main and secondary goals in a Phase 2 trial testing it for moderateto severe plaque psoriasis
ALS drug research has gained momentum from the recent approvals in the US of two new therapies Relyvrio from Amylyx options remain very limited and the available drugs benefit is modest at best for curbing ALS which causes the deterioration of motor neurons
Stockbased compensation expenses Stockbased compensation expenses represent noncash charges related to equity awards granted by the Company
They have seen a surge in industry interest particularly following approvals for Novartis drugs Lutathera and last year Pluvicto. The drugs are tricky to make and even tougher to produce at scale. Though sales of the first radiopharmaceuticals approved were modest, Pluvicto and Lutathera are proving more successful
US approval could help significantly expand use and sales revenue which amounted to 72 million through the first six months of 2023
I am honored to work with the Rett syndrome community and help patients and families suffering from this devastating disease
Gritstone company has been working on a next-generation COVID vaccine since 2021 when it partnered with the National Institutes of Health . The company aims to build a shot capable of generating both B cell and T cell immunity potentially translating to broader protection. The vaccine is designed to be selfamplifying meaning it spurs cells to create multiple copies of the encoded antigen RNA
Higher doses than the RDA should not be given to try to achieve normal serum vitamin A levels during treatment with ONPATTRO as serum levels do not reflect the total vitamin A in the body
If Royalty or PTC chooses to exercise one of the options for a bigger sale of royalties those percentages would increase
A new biotechnology company with tens of millions of dollars in funding aims to develop more precise treatments for socalled neuropsychiatric disorders
At the time analysts noted how it was rare for the FDA to call such a meeting after a rejection and rarer still for advisers to disagree with the FDAs original decision
GSK agreed to pay at least 700 million for rights to two of Alectors experimental drugs both of which target a protein that normally helps control immune system activity in the brain
NDI101150 is in a Phase 12 trial in solid tumors with results expected next year according to a federal clinical trials database . Behind NDI101150 are a handful of preclinical programs among them another cancer drug prospect as well as metabolic disease treatments being developed with Eli Lilly
Ask your healthcare provider for medical advice about side effects and about any side effects that bother the patient or that do not go away102023 805 PM US Food and Drug Administration Approves BioMarins VOXZOGO vosoritide for Children Under 5 Years with Achondroplasia  Oct 20  httpsinvestorsbiomarincom20231020USFoodandDrugAdministrationApprovesBioMarinsVOXZOGORvosoritideforChildrenUnder5Yearswith 711How is VOXZOGO taken