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GeneTx and Ultragenyx Receive Approval from U.K. Regulatory Agency to Begin Clinical Study of GTX-102 for the Treatment of Angelman Syndrome

Thursday, June 10, 2021 - 1:30pm

Medicines and Healthcare Products Regulatory Agency (MHRA)has approved the Clinical Trial Application (CTA) for the Phase 1/2 study of GTX-102 in pediatric patients with Angelman syndrome in the U.K.

Key Points: 
  • Medicines and Healthcare Products Regulatory Agency (MHRA)has approved the Clinical Trial Application (CTA) for the Phase 1/2 study of GTX-102 in pediatric patients with Angelman syndrome in the U.K.
  • Based on the outcome of the meeting, GeneTx will submit an amended protocol to FDA that addresses key FDA issues.
  • Weve made significant progress on the regulatory front over the past few weeks with the Phase 1/2 study cleared to begin enrollment in two countries.
  • In August 2019, GeneTx and Ultragenyx announced a partnership to develop GTX-102, with Ultragenyx receiving an exclusive option to acquire GeneTx.

Ultragenyx to Present at Goldman Sachs 42nd Annual Healthcare Conference

Thursday, June 3, 2021 - 9:01pm

NOVATO, Calif., June 03, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today announced that Emil D. Kakkis, M.D., Ph.D., the company's Chief Executive Officer and President, will present at the Goldman Sachs 42nd Annual Healthcare Conference on Thursday, June 10, 2021 at 3:00 PM ET.

Key Points: 
  • NOVATO, Calif., June 03, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today announced that Emil D. Kakkis, M.D., Ph.D., the company's Chief Executive Officer and President, will present at the Goldman Sachs 42nd Annual Healthcare Conference on Thursday, June 10, 2021 at 3:00 PM ET.
  • The live and archived webcast of the presentation will be accessible from the companys website at http://ir.ultragenyx.com/events.cfm .
  • Ultragenyx is a biopharmaceutical company committed to bringing to patients novel therapies for the treatment of serious rare and ultra-rare genetic diseases.
  • The company is led by a management team experienced in the development and commercialization of rare disease therapeutics.

Ultragenyx to Present at Jefferies Virtual Healthcare Conference

Wednesday, May 26, 2021 - 1:31pm

NOVATO, Calif., May 26, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today announced that Emil D. Kakkis, M.D., Ph.D., the company's Chief Executive Officer and President, will present at the Jefferies Virtual Healthcare Conference on Tuesday, June 1, 2021 at 4:30 PM ET.

Key Points: 
  • NOVATO, Calif., May 26, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today announced that Emil D. Kakkis, M.D., Ph.D., the company's Chief Executive Officer and President, will present at the Jefferies Virtual Healthcare Conference on Tuesday, June 1, 2021 at 4:30 PM ET.
  • The live and archived webcast of the presentation will be accessible from the companys website at http://ir.ultragenyx.com/events.cfm .
  • Ultragenyx is a biopharmaceutical company committed to bringing to patients novel therapies for the treatment of serious rare and ultra-rare genetic diseases.
  • For more information on Ultragenyx, please visit the company's website at: www.ultragenyx.com .

GeneTx and Ultragenyx Receive Clearance from Health Canada to Begin Clinical Study of GTX-102 in Canada for the Treatment of Angelman Syndrome

Wednesday, May 19, 2021 - 1:30pm

Patients will then move to a maintenance phase during which they will receive GTX-102 every three months and continue to be monitored for response and safety.

Key Points: 
  • Patients will then move to a maintenance phase during which they will receive GTX-102 every three months and continue to be monitored for response and safety.
  • In almost all cases of Angelman syndrome, the maternal UBE3A allele is either missing or mutated, resulting in limited to no protein expression.
  • While individuals with Angelman syndrome have a normal lifespan, they require continuous care and are unable to live independently.
  • GeneTx was launched by FAST, a patient advocacy organization and the largest non-governmental funder of Angelman syndrome research.

Ultragenyx Announces Positive Multi-Year Durability Data from Ongoing Phase 1/2 Gene Therapy Studies and Data on HeLa 3.0 Manufacturing Platform at American Society of Gene & Cell Therapy 2021 Annual Meeting

Friday, May 14, 2021 - 1:30pm

The primary endpoint is expected to be the reduction in oral glucose replacement with cornstarch while maintaining or improving glucose control assessed by continuous glucose monitoring.

Key Points: 
  • The primary endpoint is expected to be the reduction in oral glucose replacement with cornstarch while maintaining or improving glucose control assessed by continuous glucose monitoring.
  • These cell line engineering improvements result in a 2- to 5-fold yield improvement over the HeLa 2.0 platform, and a 50-fold yield improvement over HeLa 1.0.
  • It is caused by a defective gene coding for the enzyme G6Pase-\xce\xb1, resulting in the inability to regulate blood sugar (glucose).
  • It has been shown in preclinical studies to normalize levels of urinary orotic acid, a marker of ammonia metabolism.

Ultragenyx to Present at Bank of America Securities Healthcare Conference

Monday, May 10, 2021 - 9:01pm

b"NOVATO, Calif., May 10, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today announced that Mardi Dier, the company's Chief Financial Officer and Camille Bedrosian, M.D., the company\xe2\x80\x99s Chief Medical Officer, will present at the BofA Securities Healthcare Conference on Wednesday, May 12, 2021 at 3:30 PM ET.\nThe live and archived webcast of the presentation will be accessible from the company\xe2\x80\x99s website at http://ir.ultragenyx.com/events.cfm .

Key Points: 
  • b"NOVATO, Calif., May 10, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today announced that Mardi Dier, the company's Chief Financial Officer and Camille Bedrosian, M.D., the company\xe2\x80\x99s Chief Medical Officer, will present at the BofA Securities Healthcare Conference on Wednesday, May 12, 2021 at 3:30 PM ET.\nThe live and archived webcast of the presentation will be accessible from the company\xe2\x80\x99s website at http://ir.ultragenyx.com/events.cfm .
  • The replay of the webcast will be available for 90 days.\nUltragenyx is a biopharmaceutical company committed to bringing to patients novel therapies for the treatment of serious rare and ultra-rare genetic diseases.
  • The company has built a diverse portfolio of approved medicines and treatment candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.\nThe company is led by a management team experienced in the development and commercialization of rare disease therapeutics.
  • Ultragenyx\xe2\x80\x99s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.\nFor more information on Ultragenyx, please visit the company's website at: www.ultragenyx.com .\n"

Ultragenyx Reports First Quarter 2021 Financial Results and Corporate Update

Tuesday, May 4, 2021 - 9:01pm

ET to discuss the first quarter 2021 financial results and provide a corporate update.

Key Points: 
  • ET to discuss the first quarter 2021 financial results and provide a corporate update.
  • The live and replayed webcast of the call will be available through the company\xe2\x80\x99s website at https://ir.ultragenyx.com/events.cfm .
  • To participate in the live call by phone, dial (855) 797-6910 (USA) or (262) 912-6260 (international) and enter the passcode 6883837.
  • Ultragenyx undertakes no obligation to update or revise any forward-looking statements.

Ultragenyx to Present at Truist Securities Life Sciences Summit

Wednesday, April 28, 2021 - 9:01pm

b"NOVATO, Calif., April 28, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today announced that Emil Kakkis, M.D., Ph.D., will present at the 7th Annual Truist Securities Life Sciences Summit on Wednesday, May 5, 2021 at 1:00 PM ET.\nThe live and archived webcast of the presentation will be accessible from the company\xe2\x80\x99s website at http://ir.ultragenyx.com/events.cfm .

Key Points: 
  • b"NOVATO, Calif., April 28, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today announced that Emil Kakkis, M.D., Ph.D., will present at the 7th Annual Truist Securities Life Sciences Summit on Wednesday, May 5, 2021 at 1:00 PM ET.\nThe live and archived webcast of the presentation will be accessible from the company\xe2\x80\x99s website at http://ir.ultragenyx.com/events.cfm .
  • The replay of the webcast will be available for 90 days.\nUltragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of serious rare and ultra-rare genetic diseases.
  • The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.\nThe company is led by a management team experienced in the development and commercialization of rare disease therapeutics.
  • Ultragenyx\xe2\x80\x99s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.\nFor more information on Ultragenyx, please visit the company's website at: www.ultragenyx.com .\n"

Ultragenyx to Host Conference Call for First Quarter 2021 Financial Results and Corporate Update

Wednesday, April 28, 2021 - 1:31pm

b"NOVATO, Calif., April 28, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel therapies for serious rare and ultra-rare genetic diseases, today announced that it will host a conference call on Tuesday, May 4, 2021 at 5pm ET to discuss its financial results and corporate update for the first quarter ended March 31, 2021.\nThe live and replayed webcast of the call will be available through the company\xe2\x80\x99s website at https://ir.ultragenyx.com/events-presentations .

Key Points: 
  • b"NOVATO, Calif., April 28, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel therapies for serious rare and ultra-rare genetic diseases, today announced that it will host a conference call on Tuesday, May 4, 2021 at 5pm ET to discuss its financial results and corporate update for the first quarter ended March 31, 2021.\nThe live and replayed webcast of the call will be available through the company\xe2\x80\x99s website at https://ir.ultragenyx.com/events-presentations .
  • To participate in the live call by phone, dial (855) 797-6910 (USA) or (262) 912-6260 (International) and enter the passcode 6883837.
  • The replay of the call will be available for one year.\nUltragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of serious rare and ultra-rare genetic diseases.
  • Ultragenyx\xe2\x80\x99s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.\nFor more information on Ultragenyx, please visit the company's website at:\n"

Ultragenyx Announces Upcoming Data Presentations at American Society of Gene & Cell Therapy 2021 Virtual Annual Meeting

Tuesday, April 27, 2021 - 10:47pm

The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.\nThe company is led by a management team experienced in the development and commercialization of rare disease therapeutics.

Key Points: 
  • The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.\nThe company is led by a management team experienced in the development and commercialization of rare disease therapeutics.
  • Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements.
  • Ultragenyx undertakes no obligation to update or revise any forward-looking statements.
  • For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx\'s Annual Report on Form 10-K filed with the Securities and Exchange Commission on February 12, 2021, and its subsequent periodic reports filed with the Securities and Exchange Commission.\n'